HDSA Celebrates 40th Anniversary by sdfgsg234

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									                                                                                  H u nt in gton’s Dise ase Soci et y of A m e rica
                                                                                                                           November 2007

                                                                                  H un ti ngton ’s Disease Socie ty of A m e ri ca

                                                                            HDSA Celebrates 40th Anniversary


                                                                World Congress Meets in Germany
       Huntington’s Disease Society of America                  Clinical Trial Update
       505 Eighth Avenue, Suite 902
       New York, New York 10018                                 Coping with Cognitive Decline
HDSA   212-242-1968 • 1-800-345-HDSA • www.hdsa.org
                                                           a message from C. Walt Johnston                                                                                                                                                 a message from Barbara T. Boyle
                                                                                                                        HDSA’s Chairman of the Board                                                                                                                                     HDSA’s National Executive Director/CEO

        I                                                                                                                                                                         W
        Dear HDSA Community:
                am very pleased to be writing          Every dollar given, large or small, has                    us closer to the ultimate goal. A cure                                                  elcome to a                      consisting of volunteers representing                           Group clinical trial site so our families
                to you as the new Chairman of          had a positive impact on HD research                       for HD!                                                                                 new issue of                     each of our stakeholder groups selected                         have incredible opportunities to be
                the Board of the Huntington’s          and we are now just beginning to realize                                                                                                           The Marker                       revision of the guidelines that are used                        a part of the search for the missing
                                                                                                                  In the coming months, we will be
                Disease Society of America.            and appreciate the results of those                                                                                                                magazine. We                     by the Social Security Administration                           pieces of the HD puzzle. Our Centers
                                                                                                                  working with our research partners
                We wish to thank Charles               investments. As one family member                                                                                                                  are very pleased                 to determine eligibility for disability                         of Excellence also offer comprehensive
                                                                                                                  and our HDSA Centers of Excellence
        Dimmler III for his leadership and             succinctly stated – it takes money to                                                                                      to report on our progress in the areas                   benefits. We will keep you apprised                              medical and social services to those
                                                                                                                  and chapters to develop ways that
        dedication during his tenure and               conduct research – and so we thank                                                                                         of research, care and advocacy. As we                    of progress on this important issue but                         affected by HD and their families, as
                                                                                                                  we can help to ensure a vital and
        I look forward to a fruitful and               all of our donors, along with the CHDI                                                                                     close our 40th Anniversary celebration,                  you can read more about the Grassroots                          well as outreach to local long term
                                                                                                                  energized participant base. An active
        rewarding term as your new chair.              organization who have believed, as we                                                                                      we have much to celebrate and                            Technical Advocacy Grant on page 21.                            care facilities, lay and professional
                                                                                                                  participation base will allow HDSA
        It’s truly an honor!                           do, that the answers are within reach.                                                                                     commemorate. In June, the HDSA                                                                                           education, support groups and
                                                                                                                  to play an integral role in moving                                                                                       In the area of research, we continue to
                                                                                                                                                                                  convention was held in Oklahoma,                                                                                         more. Please support your Center of
        At our board meeting on October 19,            But as important as investing dollars                      compounds from the development                                                                                           move the pipeline for drug discovery
                                                                                                                                                                                  birthplace of Woody Guthrie and while                                                                                    Excellence by being a part of a walk,
        the Board of Trustees was given an             in research may be, so too is active                       stages to drug approval.                                                                                                 forward at a rapid rate. Our HDSA
                                                                                                                                                                                  we were there, we had the opportunity                                                                                    hoop or any fund raising event held
        extensive update by our researchers            participation in one of the human clinical                                                                                                                                          Coalition for the Cure investigators are
                                                                                                                  In the meantime, I thank you for your                           to meet several members of the Guthrie                                                                                   to benefit this vital care program.
        on how the dollars that you contribute         trials. CHDI is currently investigating                                                                                                                                             working to identify viable targets for
                                                                                                                  continued support and look forward                              family including Mary Jo Edgmon, the
        are being used to advance the pipeline         several compounds in assays and                                                                                                                                                     CHDI, our partner in drug discovery.                            When Marjorie Guthrie first traveled
                                                                                                                  to seeing you at the HDSA national                              “baby” sister of Woody Guthrie and
        for drug discovery. As Barbara notes           animal models. In order to achieve                                                                                                                                                  In a recent update by CHDI’s Chief                              the country in search of HD families,
                                                                                                                  convention in June.                                             several of Woody’s nieces and grand
        in her letter to you, we heard some            our goal of developing effective                                                                                                                                                    Scientific Officer, Dr. Robert Pacifici,                           she used the words “hopeless and
                                                                                                                                                                                  nieces. It was quite a festive time and
        very encouraging news from all of              treatments for HD, our clinicians will                     Sincerely yours,                                                                                                         they are in the process of testing more                         helpless”. I am very pleased that those
                                                                                                                                                                                  we were delighted when the Guthrie
        our partners and we are determined             need hundreds of HD family members.                                                                                                                                                 than 50 novel compounds that may                                words are no longer true. Today the
                                                                                                                                                                                  family agreed to join us in New York
        to find new and creative ways that              Just as our family members worked                                                                                                                                                   soon yield one or more potential                                outlook for our families has never
                                                                                                                                                                                  for our Eleventh Annual Guthrie
        we can help to fund and support this           with our scientists to help discover                       C. Walt Johnston                                                                                                         therapies for human clinical trials. With                       been more hopeful nor has the future
                                                                                                                                                                                  Awards Dinner that took place on
        essential research.                            the gene in 1993, so too will you be                       Chairman of the Board                                                                                                    your support, we can continue to fund                           held such promise. Now more than
                                                                                                                                                                                  October 18. You can read about our
                                                       called upon to help us test potentially                                                                                                                                             this essential scientific pipeline.                              ever, the answers are within reach.
        We are grateful to those of you who                                                                                                                                       stellar dinner on page 27.
                                                       effective treatments that will move
        have helped us to support this pipeline.                                                                                                                                                                                           In the area of care, our HDSA Center                            Sincerely yours,
                                                                                                                                                                                  We are also pleased to report on our
                                                                                                                                                                                                                                           of Excellence program continues to
                                                                                                                                                                                  advances in advocacy. In June, HDSA
                                                                                                                                                                                                                                           grow and to play an important part in
                                                                                                                                                                                  was awarded a $25,000 in kind grant
                                                                                                                                                                                                                                           bringing potential new treatments to                            Barbara T. Boyle
                                                                                                                                                                                  from the National Health Council to
                                                                                                                                                                                                                                           clinical trials. Every HDSA Center of                           National Executive Director/CEO
                                                                                                                                                                                  help us to build our grassroots program
                                                                                                                                                                                                                                           Excellence is also a Huntington Study
                                                                                                                                                                                  across the country. The task team,

The Marker is an official publication of the         The Marker, a periodical of the Huntington’s Disease           HDSA, Inc., is a national not-for-profit organization founded                                                                                                                  November 2007
Huntington’s Disease Society of America, Inc.       Society of America, Inc., is published twice annually.         in 1986 to help individuals with Huntington’s Disease and
505 Eighth Avenue, Suite 902
New York, NY 10018, 212-242-1968
                                                    Its purpose is to provide information and opinion and
                                                    to relay items of interest to individuals with Huntington’s
                                                                                                                   their families.

                                                                                                                   The Society is a member of the National Health
                                                                                                                                                                                        TABLE OF CONTENTS
                                                    Disease and their families, health care professionals, and
C. Walt Johnston, Chair of the Board                                                                               Council, the National Foundation for Brain Research,                                   HDSA History . . . . . . . . . . . . . . . . . . . . . . . . . . . . .2          Progress Report . . . . . . . . . . . . . . . . . . . . . . . . . . 20
                                                    interested friends and supporters.
                                                                                                                   the International Huntington Organization, the National                                HDSA Research . . . . . . . . . . . . . . . . . . . . . .3-13, 25                Advocacy . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21
Barbara T. Boyle, National Executive Director/CEO
                                                    The appearance of advertising, or the mention of               Organization for Rare Disorders, the National Voluntary
                                                                                                                                                                                                          At Risk . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .14-15     Resource Directory . . . . . . . . . . . . . . . . . . . . .22-24
Debra Lovecky, Director of Education/Editor         commercial products available for sale in articles             Health Agencies, the Alliance of Genetic Support Groups
                                                                                                                                                                                                          Feature Story: Hospice . . . . . . . . . . . . . . . . . . . . 16                HDSA 40th Anniversary . . . . . . . . . . . . . . . . .26-27
                                                    published in this publication, is not an HDSA, Inc.,           and the Independent Sector.
Karen Tarapata, Editor
                                                    guarantee or endorsement of the product or the                                                                                                        Feature Story: Tai Chi . . . . . . . . . . . . . . . . . . . . . . .17           Ways to Give . . . . . . . . . . . . . . . . . . . . . . . . . .28-30
Design: The Byne Group                                                                                             The Huntington’s Disease Society of America meets all nine
                                                    claims made for the product by the manufacturer.                                                                                                      Caregivers Link . . . . . . . . . . . . . . . . . . . . . . . . .18-19
Jennifer Zapf, Art Director                                                                                        standards of the National Charities Information Bureau.
                                                    Statements and opinions expressed in articles are
Melissa Behrens, Designer                           not necessarily those of HDSA, Inc.                            ©2007 Huntington’s Disease Society of America
                                                                                                                   Volume 9, #1
          HDSA History                                                                                                                               research
                                                                                                                                                       HDSA Research

                 HDSA Celebrates                                                                                                                            HDSA Pipeline Update
                 40th Anniversary (1967-2007)

    W                                                                                                                                          H
                 oody Guthrie was a               HD. The recommendations that resulted                                                                    DSA has an ultimate goal of         and to then screen large collections            are acceptable, the drug may then be
                 storyteller who used music       from that 1977 report have served as the                                                                 developing treatments effective     (hundreds of thousands to millions) of          approved by the Food and Drug Admin-
                 to tell his stories. His work    cornerstone of HDSA’s commitment to                                                                      in preventing or delaying the       chemical compounds to identify those            istration for treatment of the disorder.
    ranged from social commentaries about         the care and cure of HD.                                                                     onset and progression of the disorder.          that alter the results measured in the
                                                                                                                                                                                                                                               The development of effective drugs
    the working conditions of migrant workers                                                                                                  In order to expedite the movement of            assay. Promising chemical compounds
                                                  In 1983, HDSA Coalition for the Cure                                                                                                                                                         through the pipeline is a long and
    and the urban poor to ballads and                                                                                                          basic research into effective treatments,       are then retested in several different ways
                                                  investigator, Dr. Jim Gusella, found the                                                                                                                                                     expensive process, with most or all of
    children’s songs.                                                                                                                          HDSA has developed a “pipeline” of              and are subjected to slight modifications
                                                  very first marker for the disease and,                                                                                                                                                        the initial list of chemical compounds
                                                                                                                                               drug discovery that takes a systematic,         to choose a more potent structure. Many
    In 1967, Woody Guthrie, lost his battle       after a ten year search that involved                                                                                                                                                        falling away as they are subjected to
                                                                                                                                               comprehensive approach to maximizing            compounds are eliminated during this
    with HD. He was just 55 years old. During     collaboration among the top HD                                                                                                                                                               more and more stringent tests. The
                                                                                                                                               the opportunities to achieve our goal – an      testing and retesting. The compounds
    the more than 15 years that the disease       researchers worldwide, the gene                                                                                                                                                              choice of targets at the beginning of the
                                                                                                                            Marjorie Guthrie   effective treatment for HD.                     that survive are further optimized and
    affected him, he struggled to continue to     was located on the short arm of                                                                                                                                                              process is crucial to the efficient use of
                                                                                                                                                                                               then tested in animal models for possible
    communicate his conviction that every         chromosome 4.                                                                                At the beginning of each drug discovery                                                         available funds. Rigorous scientific
                                                                                                services to those affected by HD and                                                           toxicity or other reasons that might
    man, woman and child has within them                                                                                                       effort, a biological process is identified                                                       evidence supporting the choice of a
                                                  Since that time, research has progressed      their families while a toll free helpline                                                      prevent them from being used as a
    the power to make a difference.                                                                                                            as possibly essential to some critical aspect                                                   target comes from the basic research we
                                                  rapidly and, in 2004, HDSA formed a           and extensive national web site (www.                                                          drug in people. Many compounds are
                                                                                                                                               of the disease. This biological process, or                                                     support, which provides a firm foundation
    Soon after his death, his widow Marjorie      pipeline for drug discovery that begins in    hdsa.org) help to provide access to                                                            again discarded at this juncture. In the
                                                                                                                                               the protein at the center of it, is referred                                                    for HDSA’s entire drug discovery pipeline.
    vowed to do something about this silent       the laboratory with basic science (HDSA       services. HDSA chapters, affiliates,                                                            HDSA Pipeline, these steps are carried
                                                                                                                                               to as a “target” for drug discovery. In our                                                     In this issue, we feature an update on
    killer. At the time, little was known about   Grants & Fellowship program and the           regions, social workers, and support                                                           out by our partner, CHDI, Inc.
                                                                                                                                               pipeline, targets are determined through                                                        the basic research conducted by Teams
    the disease. Marjorie placed a small ad       prestigious HDSA Coalition for the            groups work in tandem with the Centers
                                                                                                                                               basic research investigations funded by         Eventually, chemical compounds that             3, 4 and 5 of the HDSA Coalition for
    in a New York City newspaper and              Cure) progresses to applied/translational     of Excellence to increase awareness
                                                                                                                                               HDSA and performed by the Coalition             survive all the tests become candidates         the Cure, a progress report on clinical
    slowly gathered a determined handful          research (HDSA partner CHDI) and then         about HD and raise funds for research,
                                                                                                                                               for the Cure and our HDSA Grants and            for testing in humans. This requires a          trials, notes on unique research taking
    of volunteers and HD families from            moves to the patient’s bedside in the         education and family services.
                                                                                                                                               Fellows Program.                                three-phase clinical trial process that,        place at our HDSA Centers of Excellence
    across the United States. From that first      form of human clinical trials that test
                                                                                                Marjorie Guthrie died just a few months                                                        in our pipeline, is carried out at sites that   and an update on the trials being
    moment, when Marjorie Guthrie reached         the most promising compounds often at                                                        Once a target has been identified, the
                                                                                                before the marker was found in 1983.                                                           include HDSA Centers of Excellence. If          conducted by Huntington’s Disease
    out to other HD families, a worldwide         HDSA Centers of Excellence. Today the                                                        next step is to devise an assay that permits
                                                                                                But in the 16 years that she worked to                                                         the drug is found effective after all three     Drug Works (HDDW). ■
    movement began that would change the          question our families ask is not “if” there                                                  reproducible measurement of the target
                                                                                                bring this disease out of the family closet,                                                   phases of testing, and any side effects
    lives of those living with HD and bring       will be a treatment or cure, but “when.”
                                                                                                she brought empathy and hope – a hope
    hope to families.
                                                  In the area of care, HDSA has created         for a future free of HD – to those affected
    Dynamic and compelling, Marjorie              a national network of resources and           by this devastating disease. Her work
                                                                                                                                                               The HDSA Research Pipeline
    Guthrie convinced then President Jimmy        referrals that are unmatched by any           resulted in what is today the Huntington’s
    Carter to form a Presidential Commission      other HD organization. HDSA Centers           Disease Society of America. And so we
                                                                                                                                                                      Step 1                       Step 2-4                                 Step 5-7
    to study neurological diseases, including     of Excellence provide medical and social      pay tribute to Marjorie Guthrie in this
                                                                                                                                                                  Basic Research               Applied Research                    Phase 1, 2, 3 Clinical Trials
                                                                                                issue for her unflagging efforts to educate,
                                                                                                empower and encourage those affected                            Molecular                                                                               FDA Approved
                                                                                                by HD, their families and the many                              Target                                                                                          Drug
    SAVE THE DATE                                                                               researchers around the world. ■
    HDSA Annual Convention • June 6-8, 2008                                                                                                                        HDSA Coalition                HDSA Partnership                 HDSA Partnership for Clinical Trials
                                                                                                                                                                   for the Cure                  with CHDI
    Omni William Penn Hotel • Pittsburgh, PA                                                                                                                                                                                      HDSA Centers of Excellence
                                                                                                                                                                   HDSA Grants and
    Go to www.hdsa.org for more information after January 1, 2008.                                                                                                 Fellows Program

2     WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                               Huntington’s Disease Society of Amer ica

                                                                                                                                                                                                                                                                           H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
           HDSA Research                                                                                                                                     research
                                                                                                                                                               HDSA Research

                 5th Annual Phyllis E. Dake
                                                                                                                                                       whether other cellular changes occurred
                                                                                                                                                       first. Their findings indicated that the

                 Huntington’s Disease Research Symposium                                                                                               problem in HD is not a defect in the
                                                                                                                                                       mitochondria’s capacity to produce energy.
                                                                                                                                                       At the same time, the Gusella group

                                                                                                                                                       investigated variations in mitochondrial
             he annual Phyllis E. Dake               pipeline the cost of their development           and how does this contribute to disease
                                                                                                                                                       DNA that might correlate with HD, but
             Huntington’s Disease Research           increases exponentially. Thus, identifying       pathogenesis? Members of the same
                                                                                                                                                       did not find any evidence of a correlation.
             Symposium serves two functions:         outstanding targets in the basic research        team may be working on very different
                                                                                                                                                       Dr. MacDonald’s group also compared
    first, it provides an opportunity to hear         phase is crucial given the large financial        aspects of a question. The importance
                                                                                                                                                       the response of cells expressing mutant
    the latest Huntington Disease (HD)               investment necessary in subsequent               of the Coalition is that it hastens progress
                                                                                                                                                       huntingtin with cells in which the
    research from leading scientists and how         stages. The Coalition for the Cure and           by encouraging collaboration and the
                                                                                                                                                       mitochondria has been poisoned
    it will lead to therapies, and secondly,         the Grants & Fellows program provides            sharing of materials to accelerate research.
                                                                                                                                                       chemically. The results suggest that mutant                                                 Nora Riley with Perrin Dake and Barbara Boyle
    to recognize and honor Phyllis Dake,             funding for this essential basic research.
                                                                                                      Dr. Gusella is part of Team 5, the               huntingtin affects genes involved in
    a longtime friend to the HD research
                                                     HDSA Coalition for the Cure researchers          Normal Function Team, which seeks                gluconeogenesis and lipid metabolism,          which inhibit various protease families,     Dr. Jang-Ho Cha is a member of Team 4,
    community. Although, Mrs. Dake could
                                                     have worked in teams, whose goals are            to understand the role and behavior of           but not specifically genes affected             were screened for their ability to prevent   the Transcription Team. The goal of this
    not attend, “she sends her very best and
                                                     to answer one of five basic questions             normal huntingtin protein and how this           by mitochondrial dysfunction. Dr.              small fragments of mutant huntingtin         team is to understand how mutant
    hopes to be there next year.”
                                                     about HD: 1) Is the problem in HD an             may contribute to disease prevention.            MacDonald has found that the                   from being produced in a striatal cell       huntingtin disturbs these processes and to
    Barbara Boyle, HDSA National Executive           inherent defect in mitochondrial energy          His laboratory is seeking to understand          polyglutamine tract in both normal             model. One inhibitor of these protease       apply this knowledge to the development
    Director/CEO, opened the symposium               metabolism?; 2) How does huntingtin              the disruption of a normal function of           and mutant huntingtin correlates with          families was found to be effective in        of new therapies. He discussed the
    by welcoming the panel of scientists and         aggregation impact Huntington’s Disease          huntingtin which may drive pathology             cellular energy levels. She believes that      preventing formation of the smallest         importance of a multi-pronged
    guests. Jim Gusella, HDSA Coalition              development and pathogenesis?; 3) What           in HD. In these experiments, the gene            huntingtin is like a giant “slinky” that       fragment (named cpA). Dr. DiFiglia’s         approach to developing HD therapies.
    for the Cure investigator, from the              are the post-translational modifications          encoding huntingtin was disrupted in             can act as a scaffold for various functions    work was performed as part of a drug         Transcriptional dysfunction repeatedly
    Center for Human Genetic Research                that huntingtin undergoes and how can            Drosophila melanogaster (fruit flies) and         within a cell, but that has no intrinsic                                                    arises as an important target for potential
    at Massachusetts General Hospital,               interruptions of this alter pathogenesis?;       also in Dictyostelium discoideum (slime          enzymatic activity. Drs. Gusella and           “The research supported                      therapies, as huntingtin interacts
    explained the discovery pipeline used            4) What are the primary signaling                mold) creating “knock-out” systems. For          MacDonald are also collecting samples                                                       with many proteins that function in
    to take candidate therapies from an idea         pathways through which huntingtin acts           fruit flies, it was found that huntingtin is      for a large Age of Onset genetic modifier       by HDSA and presented                        transcriptional regulation. Because
    to a success in the clinic (see page 3           and what is its impact on transcription          not a necessary protein. For slime mold,         screen using motor symptoms as a
    for a full description of the pipeline). As      regulation?; and 5) What is the role and         however, huntingtin is necessary for the
                                                                                                                                                                                                      at the Dake Research                         different genes are being actively
                                                                                                                                                       phenotypic indicator. This screen will                                                      transcribed in different cells types,
    potential therapies move through this            behavior of normal huntingtin protein            organism to complete its life cycle. This        assess over 1 million spots across the         Symposium is expanding                       this could explain why neurons are
                                                                                                      system may therefore yield information           entire genome to find normal variations                                                      specifically affected whereas other cell
                                                                                                      regarding a normal function of huntingtin        that can alter the course of HD.               our understanding of the                     types that also express mutant huntingtin
                                                                                                      as its rapid lifecycle is suitable for quickly
                                                                                                                                                       Dr. Marian DiFiglia is a member of Team        basic processes of HD.”                      are not. While the task of fixing complex
                                                                                                      testing genetic modifiers and drugs.                                                                                                          transcriptional abnormalities may seem
                                                                                                                                                       3, the Proteolysis and Post-translational
                                                                                                      Dr. Marcy MacDonald is also part of the                                                         discovery program developed by Anne          daunting, in fact therapies to attain this
                                                                                                                                                       Modification Team. This team conducts
                                                                                                      Normal Function Team. Both she and                                                              Young at MassGeneral Institute for           goal are already being implemented in
                                                                                                                                                       basic science on the huntingtin protein
                                                                                                      Dr. Gusella were also a part of Team 1,                                                         Neurodegenerative Disease (MIND) in          the cancer field.
                                                                                                                                                       to determine whether the cleavage
                                                                                                      the Mitochondrial Energy Metabolism,                                                            collaboration with Novartis. The next
                                                                                                                                                       of huntingtin is a key factor in the                                                        Dr. Steve Hersch, also a member of the
                                                                                                      but in the past year that Team achieved                                                         step is to test these compounds in a
                                                                                                                                                       development of the disease and then                                                         Transcription Team, is testing compounds
                                                                                                      an answer to its fundamental question.                                                          rapid, in vivo mouse model that Dr.
                                                                                                                                                       uses this knowledge to define therapeutic                                                    identified by basic research in animal
                                                                                                      Dr. MacDonald’s group performed                                                                 DiFiglia has created. Small huntingtin
                                                                                                                                                       strategies. Cleavage of mutant huntingtin                                                   models, including those that bolster energy
                                                                                                      experiments to address whether the first                                                         fragments are produced and accumulate
                                                                                                                                                       can create small fragments which can                                                        production and modify transcriptional
                                                                                                      changes observed in cells expressing                                                            in the mice allowing one to test the
                                                                                                                                                       accumulate in striatal brain cells, travel                                                  activities. The team is working with
                                                                                                      mutant huntingtin were changes relating                                                         efficacy of these inhibitors to block cpA
                                                                                                                                                       to the cell’s nucleus and disrupt the cell’s
                                                                                                      to mitochondria energy function or                                                              in vivo and determine whether this has       —continued on page 32.
            Barbara Boyle with Coalition investigators Marian DiFiglia, Steven Hersch, Jang-Ho Cha,                                                    function. About 100 small molecules,
                                                               Marcy MacDonald and Jim Gusella.                                                                                                       a beneficial effect.

4     WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                                     Huntington’s Disease Society of America

                                                                                                                                                                                                                                                                                 H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
           HDSA Research                                                                                                                        research
                                                                                                                                                  HDSA Research

                 Clinical Trial Update                                                                                                                                              PREDICT-HD began in September                        employment and insurance, and also
                                                                                                                                                                                    2001 to study healthy persons who are                from foreign sites where health care systems

                 The Huntington Study Group                                                                                                                                         known to have the CAG expansion in
                                                                                                                                                                                    the HD gene. The intention of the study
                                                                                                                                                                                                                                         may be different from domestic sites.

                                                                                                                                                                                    is to gather essential information on the            Upcoming Trials
                                                                                                                                                                                                                early stages of
    At the end of the HDSA pipeline                                                                                                                                                                                                      NIH approved funding for the 2CARE
                                                                                                                                                                           “Efforts are focused on HD in order to                        study of coenzyme Q10 versus placebo
    for drug discovery are clinical trials                                                                                                                                                                      develop drugs
    which bring the most promising                                                                                                                                         finding treatments that               that can slow
                                                                                                                                                                                                                                         in individuals with mild to moderate
                                                                                                                                                                                                                                         Huntington’s Disease. The initiation of
    compounds for treatment to                                                                                                                                             make a difference for                or postpone
                                                                                                                                                                                                                                         the study was delayed by the FDA who
    people with HD. HD families are                                                                                                                                                                             the onset of
                                                                                                                                                                                                                                         requested additional animal testing since
    encouraged to participate in the                                                                                                                                       Huntington’s Disease.” the disease.
                                                                                                                                                                                                                                         the 2CARE study is investigating a much
                                                                                                                                                                                                                To date 992
    many clinical and observational                                                                                                                                                                                                      higher dose (2400 mg./day). With the
                                                                                                                                                                                    participants have enrolled in the study.
    trials that are conducted at HDSA                                                                                                                                                                                                    additional testing completed, the FDA
                                                                                                                                                                                    Gene negative participants can still
    Centers of Excellence and other                                                                                                                                                                                                      has now given permission for the study
                                                                                                                                                                                    enroll in this important project.
                                                                                                                                                                                                                                         to proceed. Enrollment of participants at
    HSG sites.
                                                                                                                                                                                         COHORT is a long-term observational             approximately 46 sites in North America
                                                                                                                                                                                         study taking place at 40 North American         and Australia should begin in early 2008.

                                                                                                                                                                                         and Australian HSG sites. The goal of
           he HSG continues to be very                                                                                                                                                                                                   PREQUEL is a trial to test the tolerability
                                                                                                                                                                                         COHORT is to collect information from
           active in the Huntington’s Disease                                                                                                                                                                                            and biological activity of coenzyme Q10
                                                                                                                                                                                         individuals who are affected by HD and
           research arena. Efforts are focused                                                                                                                                                                                           in those with pre-manifest HD. A grant
                                                                                                                                                                                         those who are part of an HD family, in
    on finding treatments that make a                                                                                                                                                                                                     was submitted in July 2007 to the National
                                                                                                                                                                                         order to learn more about HD, potential
    difference for HD. HSG research trials                                                                                                                                                                                               Institute of Neurological Disorders and
                                                                                                                                                                                         treatments, and to plan future research
    cover a wide spectrum within the                                                                                                                                                                                                     Stroke (NINDS). PREQUEL has received
                                                 PHEND-HD completed enrollment of             prolongs life. In a previous HSG trial,     Trials Currently Enrolling                     studies of experimental drugs aimed
    Huntington’s Disease community. The          58 study participants in January 2006.       minocycline was shown to be safe and                                                                                                       positive feedback and plans are being
                                                                                                                                          DIMOND B is a clinical study using             at postponing the onset or slowing the
    following overviews describe the most        The study was designed to gather             tolerable in HD patients at doses of 100                                                                                                   finalized with NINDS. Christopher
                                                                                                                                          a research medication, Dimebon, in             progression of HD. This study is recruiting
    current HSG research trial activity.         information on the safety and tolerability   mg. and 200 mg. per day over 8 weeks                                                                                                       A. Ross, M.D., Ph.D., is the principal
                                                                                                                                          patients who have mild to moderate             both adults and children who have
                                                 of the experimental drug phenylbutyrate.     of use [Neurology 2004: 63:547-549].                                                                                                       investigator and Kevin M. Biglan, M.D.,
                                                                                                                                                                                         clinically diagnosed HD and adults who
    Trials Recently Completing                   This compound inhibits histone
                                                                                                                                          Huntington’s Disease. Dimebon is an                                                            M.P.H., is the co-principal investigator.
    Enrollment                                                                                TREND-HD was designed to determine          investigational medication that has been       are a part of an HD family. Individuals
                                                 deacetylase (HDAC), an essential                                                                                                                                                        CREST-E, a trial to test the safety,
                                                                                              the effect of the experimental medication   shown to inhibit brain cell death in models    who choose to participate will have one
    TETRA-HD completed enrollment                component of the body that “turns on”                                                                                                                                                   tolerability and efficacy of creatine in
                                                                                              ethyl-EPA on the motor (movement) signs     of HD and Alzheimer’s disease (AD).            study visit every year. There are currently
    of 84 participants in December 2003.         or stimulates genes. Phenylbutyrate may                                                                                                                                                 persons with HD, is scheduled to move
                                                                                              and symptoms of HD. 41 North American       In a 12-month study of patients with           864 participants enrolled in COHORT.
    Tetrabenazine is a drug widely available     be a potential drug therapy in HD by                                                                                                                                                    ahead. As of November 2007, funding
                                                                                              HSG research sites evaluated 316            Alzheimer’s, Dimebon was well-tolerated
    in Europe and Canada. It eases one of        directly helping to turn on genes that are                                                                                              RESPOND-HD is an observational trial
                                                                                              participants with early signs of HD.        and significantly improved cognition and                                                        has been secured from the National
                                                 blocked by HD. The potential benefit of                                                                                                  looking at issues of potential discrimination
    the most disabling symptoms of                                                            Preliminary results announced in April                                                                                                     Center for Complementary and Alternative
                                                                                                                                          memory, behavior, activities of daily living   in those affected by HD. The information
    Huntington’s Disease, involuntary            phenylbutyrate in people with HD has                                                                                                                                                    Medicine (NCCAM) and the FDA
                                                                                              2007 showed no statistically significant     and overall function when compared to
                                                 never been established. The analysis of                                                                                                 gathered in this study will allow researchers
    writhing movements known as chorea,                                                       difference between those taking the                                                                                                        Orphan Products Division. Steven
                                                                                                                                          placebo. The current HSG study will assess     to examine the experiences of persons
    according to a study published in February   the data is ongoing.                                                                                                                                                                    Hersch, M.D., Ph.D. is the Principal
                                                                                              experimental drug and those on placebo      the effects of Dimebon on cognition,           who have undergone genetic testing for
    2006 [Neurology 2006;66:366-372].            DOMINO completed enrollment of 114           over six months of study. There was no                                                                                                     Investigator.
                                                                                                                                          memory, behavior, and motor function           HD or those persons who are at risk for
    Prestwick Pharmaceuticals, Inc., the         participants in May 2007. Participants       evidence in the preliminary analysis        of HD patients, as well as the safety and      HD. Study participants from PHAROS
    study sponsor, has filed a New Drug                                                        of any major safety concern. Further                                                                                                       For more information on these studies and
                                                 are still undergoing study visits. Recent                                                tolerability of the drug when administered     and PREDICT research studies are being          how to participate, please see the HSG
    Application with the FDA to market the       studies in a mouse model of HD have          evaluation of the data is currently         over a 3-month period. DIMOND B                recruited for this study as are those from      website (www.Huntington-Study-Group.org).
    product for treatment of chorea in the       demonstrated that minocycline helps to       taking place.                               is seeking to enroll approximately 90          geographical areas where there are
    United States. Review of the application     slow down the clinical onset of HD and                                                   study participants.                            differing discrimination laws for
    is pending.

6     WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                          Huntington’s Disease Society of America

                                                                                                                                                                                                                                                                      H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
           HDSA Research                                                                                                                        research
                                                                                                                                                  HDSA Research

                 Methodology & Observations
                                                                                                                                          psychiatric events occurred. Thereafter
                                                                                                                                          motor skills were stable while cognitive

                 From the Huntington’s Disease Drug Works Trials                                                                          skills declined.

                                                                  By LaVonne Goodman, M.D., and Nathan Goodman, Ph.D.                     Non complaint early to middle
                                                                                                                                          stage participants: Six participants

              s a physician caring for            After Institutional Review Board (IRB)     Participants: Thirty participants were       were non compliant, or did not take
              Huntington’s Disease (HD)           approval, each participant obtained        enrolled over the three years. Twenty-       supplements as directed. All participants
              patients, I am often asked about    a family member’s support and local        three have been followed for between 2       in this group declined in motor,
    supplements that have shown benefit            physician’s supervision. Each performed    and 3 years (depending on individual start   cognitive, or both test measures.
    in animal models of HD. Many people           finger-tapping (motor) and cognitive        date). Of the remaining 7 participants,
                                                                                                                                          Advanced stage participants:
    use these supplements without guidance        (thinking) tests on their home computer;   there has been 1 death (due to lung
                                                                                                                                          The remaining 8 participants had
    or supervision. Even when the doctor is       first to establish individual baseline      cancer), 3 nursing home placements,
                                                                                                                                          advanced disease, or could not function
    aware of supplement use, it is difficult to    measures, and then to monitor progress     and 3 withdrawals (associated with
                                                                                                                                          independently at the beginning of the
    monitor its effect. In 2004, I established    after beginning supplements. This test     psychiatric events). They include men
                                                                                                                                          trial. Despite compliance maintained
    the Huntington’s Disease Drug Works           data, which was performed several times    and women between the ages of 34 and
                                                                                                                                          with the help of dedicated caregivers,
    (HDDW) with Nathan Goodman, a                 per month was received over the internet   75 from all areas of the country, and
                                                                                                                                          6 out of 8 participants have declined.
    computer scientist, in an effort to address   and analyzed at HDDW’s secure site.        unlike traditional clinical trials, late
                                                                                                                                          Three compliant participants were
    this situation. Together, we developed a      Functional status was independently        stage participants were not excluded.                                                      worsening. Fourth, greater benefit            to use supplements, and some don’t.
                                                                                                                                          stable, then began decline (going from
    web based testing method to monitor the       assessed by interview with each patient                                                                                               correlated with stage of disease: those      It is the patient’s choice. ■
                                                                                                                                          walking to wheelchair) at the time of
    motor and cognitive skills of volunteers      and/or family mentor.                      Results                                                                                    with earliest stage improved, middle
                                                                                                                                          medical illness or surgery. Both non                                                       About the author: LaVonne Goodman is a
    who agreed to be observed while each                                                                                                                                                stage stabilized, and late stage worsened.
                                                  Supplements: Supplements included          Compliant early to middle stage              compliant patients worsened.                                                               Huntington’s Disease family member and
    was taking a cocktail of supplements.                                                                                                                                               Though we admit there could be other
                                                  creatine, coenzyme Q10, an omega 3                                                                                                                                                 internal medicine physician in Washington
                                                                                             participants: Six out of 9 participants                                                    explanations, all of these observations      State where she cares for HD patients
    Now that the trials have reached the          combination of EPA and DHA, trehalose,     in this group who have taken all supple-     Discussion                                    fit the pattern that would be expected        and facilitates support groups. In addition
    three year mark, we are pleased to share      and blueberry extract. Except when         ments rigorously have shown improved                                                                                                    to her work with HDDW trials, she is
                                                                                                                                          We acknowledge that HDDW observa-             if all or part of the treatment cocktail
    our observations. To summarize; the           products were limited, participants were   or stable test measures of cognitive and                                                                                                editor for the HDDW website (www.
                                                                                                                                          tional trials have limitations that include   gives benefit.                                hddrugworks.org) that provides information
    majority of those with early or middle        offered all of the supplements. Care was   motor function and functional capacity.
                                                                                                                                          small numbers, no placebo controls, no                                                     on treatments for HD. She is a former
    stage disease who consistently took the       taken to supply quality supplements,       Two other participants in this group                                                       We also observed that our web based
                                                                                                                                          blinding, and possible compliance bias                                                     president of the HDSA Northwest Chapter,
    combination of supplements showed             and when possible from companies           who did not take trehalose had either                                                      test results are in close agreement to       recipient of the 2006 Hunt-Dis Hero award
                                                                                                                                          (compliant patients often have healthier                                                   and has served on the planning committee
    either improvement or stabilization of        whose product was approved for use         motor or cognitive worsening. The final                                                     the clinical outcome of every participant
                                                                                                                                          life-styles than non compliant patients).                                                  for the 2007 Inaugural HD Symposium.
    test measures and functional capacity         in FDA sanctioned clinical trials (see     participant in this group demonstrated                                                     over time. We suggest that this type of
                                                                                                                                          These limitations prevent generalizing
    during the duration of the trial.             table below).                              stability with all test measures until                                                     testing is an economical way to monitor
                                                                                                                                          of our results to the broader population
                                                                                                                                                                                        real world patients on a much larger
                                                                                                                                          of patients.
    Trial Description                                                                                                                                                                   scale than possible with present
                                                                                                                                          However, despite these limitations we         research methodology.
    HDDW trials are not conducted like              Supplement               Dose                           Source                        believe there are useful observations.
    traditional research trials. HDDW trials                                                                                                                                            And finally, as a physician treating HD
                                                    Creatine                 5 gm 2 times/day               Avicena                       First, there were no significant side
    were not placebo controlled or blinded,                                                                                                                                             patients, how do I use these results? I
                                                                                                                                          effects due to the supplement cocktail.
    which means that every participant                                                                                                                                                  give information about our observations
                                                    Coenzyme Q10             150 gm 5 times/day             Tischon                       Second, though we can’t exclude chance
    received a known supplement                                                                                                                                                         and emphasize that more certain
                                                                             (= to 1200 Vitaline/day)                                     events, motor and cognitive skills either
    combination, and unlike traditional                                                                                                                                                 answers regarding supplement use are
                                                                                                                                          improved or showed 2-3 year stabilization
    research trials, participants were allowed      Trehalose                25 gm 3 times/day              Sweet & Low                                                                 unknown until definitive clinical trials
                                                                                                                                          (or lack of progression) for the majority
    to take additional supplements or                                                                                                                                                   are completed. If a patient decides to
                                                                                                                                          of compliant early and middle stage
    medications like minocycline and DHA,           Omega 3                  1 gm EPA 2 times/day           Omega Brite                                                                 use supplements, I give guidance and
                                                                             300 mg DHA 2 times/day                                       participants. Third, participants who
    or psychiatric medications as needed.                                                                                                                                               information on safest products, and
                                                                                                                                          were non compliant or stopped
    Those with a psychiatric history were           Blueberry Extract        = to 1 cup blueberries         Brownwood Acres                                                             monitor for side effects. Some choose
                                                                                                                                          supplements during the trial showed
    not excluded.

8     WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                     Huntington’s Disease Society of America

                                                                                                                                                                                                                                                                 H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
         HDSA Research                                                                                                                           research
                                                                                                                                                   HDSA Research

              World Congress                                                                                                                Ira Shoulson (HDSA Center of
                                                                                                                                            Excellence at the University of Rochester
                                                                                                                                                                                        Jane Paulsen presented a study on
                                                                                                                                                                                        obsessive-compulsive symptoms in
                                                                                                                                                                                                                                       Elizabeth Howard (UK) presented
                                                                                                                                                                                                                                       an update on a 2003 study that used

              on Huntington’s Disease Meets in Germany                                                                                      and Chair of the Huntington Study
                                                                                                                                            Group) discussed recently completed
                                                                                                                                                                                        pre-symptomatic HD, noting that these
                                                                                                                                                                                        symptoms may be useful measures to
                                                                                                                                                                                                                                       walking and drinking time to standardize
                                                                                                                                                                                                                                       motor assessment in HD. Researchers
                                                                                                                                            and currently recruiting trials for HD.     track disease progression and important        found a significant correlation between

                                                                                                                                            Information about these studies can         contributors to functional status.             these measures and chorea and
         he third World Congress on
                                                                                                                                            be found on the Huntington Study                                                           dystonia scores.
         Huntington’s Disease met in                                                                                                                                                    Stefan Kloppel (UK) discussed
                                                                                                                                            Group website at www.huntington-
         Dresden in early September,                                                                                                                                                    irritability, depression and anxiety           Diana Rosas (New England HDSA
 bringing together the top HD investigators                                                                                                                                             in pre-symptomatic HD, finding that             Center of Excellence) presented on the
 from the World Federation of Neurology                                                                                                     Monica Busse (UK) spoke on the              pre-symptomatic individuals became             use of neuroimaging as a way to identify
 Research Group on HD and the                                                                                                               effects of physical therapy on movements    very irritated with people close to them       the factors that influence cortical thinning
 International Huntington Association                                                                                                       and function in HD and the need for         and that this irritation was related to        and subcortical atrophy, which may
 for three days of scientific, clinical and                                                                                                  studies to measure its impact.              impulse control and anger.                     account for why HD progresses differently
 lay presentations.                                                                                                                                                                                                                    in each person. Dr. Rosas also presented a
                                                                                                                                            Anne Young (Massachusetts General           Ferdinando Squitieri (Italy) discussed
                                                                                                                                                                                                                                       study on the use of Plasma 8-OH2dG as
 The Congress opened with a moving                                                                                                          Hospital) discussed how the addictive       Juvenile HD and how an expanded CAG
                                                                                                                                                                                                                                       a biomarker for HD. There are dramatic
 tribute to Milton Wexler by his daughter                                                                                                   tendencies seen in people with HD           repeat may be more toxic than an adult
                                                                                                                                                                                                                                       increases of 8-OH2dG in people with HD
 Alice, followed by an overview of the                                                                                                      could be due to an excess production        expansion, causing a more severe
                                                                                                                                                                                                                                       and elevated levels in pre-symptomatic
 disease and its history by Alice and                                                                                                       of dopamine caused by the disease.          disease course with faster loss of
                                                                                                                                                                                                                                       individuals which then spike at
 Nancy Wexler and a discussion of future                                                                                                                                                independence.
                                                                                                                                            Erik van Duijn (Netherlands) presented                                                     disease onset.
 trends led by Jim Gusella, who noted
                                                                                                                                            a study showing that pre-symptomatic        A. Painold (Austria) used a low resolution
 that there is a need for greater emphasis                                                                                                                                                                                             Kevin Biglan (University of Rochester)
                                                                                                                                            individuals have a higher incidence of      brain electromagnetic tomography
 on the entire HD disease process rather                                                                                                                                                                                               continued the discussion of 8-OH2dG
                                                                                                                                            depression and obsessive-compulsive         (LORETA) or 3D tomography to identify
 than only its end stages in order to promote                                                                                                                                                                                          by presenting the results of the Pre2Care
                                                                                                                                            disorders than those with HD, postulating   predominantly involved brain regions
 effective drug discovery.                                                                                                                                                                                                             study which, using 2400 mg./day of
                                                                                                                                            the need for adequate treatment for         in individuals with HD. Findings were
                                                                                                                                                                                                                                       CoQ10, showed no difference in levels of
 Leslie Thompson (HDSA Coalition                                                                                                            psychiatric disorders in these groups.      consistent with the known deterioration
                                                Breakout sessions followed and focused        Diana Rosas (New England HDSA                                                                                                            8-OH2dG between individuals with HD
 for the Cure) spoke on potential targets                                                                                                                                               and atrophy of the frontal lobe in HD.
                                                on studies in drug discovery and clinical     Center of Excellence) talked about the        Julie Snowden (UK) discussed a study                                                       and control participants using CoQ10.
 for treatments for HD, including the
                                                trials, clinical features of HD, biomarkers   use of neuron-imaging to measure              that assessed the difficulty people with
 165 potential proteins found by HDSA                                                                                                                                                                                                  —continued on page 31.
                                                in HD, genetic testing, and HD-related        atrophy and shrinkage in the striatum         HD have in recognizing emotions,
 Coalition for the Cure investigator
                                                disorders.                                    and cortex in order to determine who          particularly anger.
 Eric Wanker and the 17 different
 amino acids identified by HDSA                                                                would most benefit from a clinical trial.      Susie Henley (UK) presented a study
                                                Jane Paulsen (HDSA Center of
 Grant recipient Ray Truant.                                                                  Dr. Rosas also noted the results of a phase   that used MRI scans which found that
                                                Excellence at the University of Iowa)
                                                spoke about the early results of PREDICT,
                                                                                              two trial of creatine (CREST) which           an HD test group was statistically                     Centers of Excellence: Hot Topics
 Ron Wetzel (University of Pittsburgh)                                                        showed the significant impact creatine
                                                a study designed to characterize the early                                                  significantly worse than the control
 told of the varied approaches that are                                                       has on the rate of change in the cortex.                                                                • The HDSA Center of Excellence          • The HDSA Center of Excellence
                                                neuron-cognitive measures of HD for use                                                     group in recognizing emotions, with a                       at Johns Hopkins University/             at Indiana University School
 being studied to combat aggregation
                                                in clinical trials. The study is seeking      Robert Pacifici (CHDI, Inc.) elaborated        disproportionate impairment in anger                        Hospital is conducting a study           of Medicine is participating in
 within cells, stimulate the removal of
                                                renewal with the NIH.                                                                       recognition in early HD.                                    to determine whether the drug            RESPOND, an ethical, legal and
 aggregates, and block the downstream                                                         on the pipeline for drug discovery that
                                                                                                                                                                                                        mementine (Namenda) which                social implications study funded as
 effects of aggregation.                                                                      investigates unique compounds that            Julie Stout discussed findings of the                        is approved by the FDA for               an add on to the PREDICT study.
                                                Julie Stout (HDSA Center of Excellence
                                                                                              could potentially translate into treatments   PREDICT HD observational trial on the                       Alzheimer’s Disease, might also          RESPOND will collect data similar
 Michael Levine (UCLA) looked at                at Indiana University) discussed a series
                                                                                              for HD. To date, 60 molecules have                                                                        improve memory and thinking              to that collected in PHAROS for the
                                                of tests, or “toolkit”, being developed to                                                  elevated levels of depression in pre-HD
 neuronal dysfunctions in the striatum and                                                    resulted in a positive methodology that                                                                   ability in patients with HD. Go to the   purposes of comparison as well
                                                detect subtle changes in pre-symptomatic                                                    individuals, which will assist clinicians                   website at www.hopkinsmedicine.          as survey potential discrimination
 cortex that precede neurodegeneration in                                                     can be reproduced.
                                                individuals, for use in determining drug                                                    in identifying those who may benefit                         org/psychiatry/neurobiology/bhdc.        resulting from genetic testing.
 mouse models which may be precursors
                                                effects in clinical trials.                                                                 from treatment.                                             html for more information.
 of symptoms.

10 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                            Huntington’s Disease Society of America

                                                                                                                                                                                                                                                                     H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
        HDSA Research                                                                                                                         research
                                                                                                                                                HDSA Research

             Coping with Cognitive Decline in HD                                                                                        Executive functioning helps us plan,
                                                                                                                                        organize, sequence, and inhibit ourselves
                                                                                                       By Kevin Duff, M.D.              when necessary. Without these skills,
                                                                                                                                        we might have difficulties with problem
                                                                                                                                        solving (e.g., following instructions to

                                                                                                                                        baking a cake or work a DVD player).
          long with involuntary movements   Processing speed is an indicator of          information that you can process. In a
                                                                                                                                        Unfortunately, difficulties with executive
          and psychiatric symptoms,         how quickly a person can mentally and        research lab, working memory can be
                                                                                                                                        functioning can occur early in the course
          cognitive difficulties are a       physically perform specific actions.          assessed by asking someone to remember
                                                                                                                                        of HD. Ways to cope with executive
 common obstacle faced by patients with     Playing tic-tac-toe, figuring out a tip at    a long string of numbers. In day-to-day
                                                                                                                                        dysfunction include: 1) be concrete
 Huntington’s Disease (HD) and their        a restaurant, or deciding which clothes      life, working memory is tapped when we
                                                                                                                                        (i.e., don’t talk about things in the abstract);
 families. These cognitive problems         to wear all require processing speed. As     try to do more than one thing at a time
                                                                                                                                        2) be clear (i.e., say what you mean); and
 can include slowed processing speed,       HD progresses, processing speed slows        (e.g., talk on the phone and sort laundry
                                                                                                                                        3) be detailed (i.e., don’t be ambiguous).
 decreased working memory, impairments      and slows. This can be frustrating for       and pay bills). In HD, individuals lose
                                                                                                                                        For example, if you want your spouse
 in learning and memory, and executive      the individual and those around him/         this ability to “multi-task.” We can cope
                                                                                                                                        with HD to wear the blue shirt and tan
 dysfunction. Recent research indicates     her. Possible solutions for this slowed      with this decrease in working memory
                                                                                                                                        pants to the party, then say that
 that subtle cognitive problems can         processing speed include: 1) allowing        by: 1) keeping things simple (e.g., one or
                                                                                                                                        concretely, clearly, and with detail.
 begin before HD diagnosis and progress     extra time in the schedule (i.e., don’t be   two things vs. three or more); 2) writing
 throughout the course of the illness.      in a hurry or wait until the last minute);   things down (e.g., lists of errands); and      Individuals with executive difficulties
                                                                                         3) minimizing distractions (e.g., the more     also thrive with routine, so plan regular          medications that are more suitable to the        About the author: Kevin Duff is a clinical
                                            2) focus more on accuracy than speed;
 These cognitive difficulties can lead to                                                                                                                                                   specific cognitive deficits in HD are              neuropsychologist and Assistant Professor
                                            3) use closed-ended questions with           noise around you, the more strain on           activities and stick with them to make                                                              of Psychiatry at the University of Iowa. His
 day-to-day problems with work, driving,                                                                                                                                                   currently being investigated.
                                            limited choices (e.g., “Would you like       your working memory).                          life predictable (e.g., mealtimes, outings,                                                         clinic interests include neuropsychology
 and home life. Although patients and                                                                                                                                                                                                       and dementia, and his research interest
                                            chicken or fish?” vs. “What do you want                                                      doctor’s appointments). Lastly, despite a          Cognitive problems do occur with
 their families might not be able to stop                                                Similar to Alzheimer’s disease, individuals                                                                                                        includes Mild Cognitive Impairment and
                                            on this 6-page menu?”); and 4) keep                                                         great routine, changes are unavoidable.            HD. However, options are available
 the development of these cognitive                                                      with HD can appear to have difficulties                                                                                                             HD. When not at work, he enjoys spending
                                            conversations focused on one topic.                                                         If unexpected changes need to occur,               to cope with these cognitive problems            time with his family outdoors and riding
 difficulties, there are some things they                                                 learning and recalling information.
                                                                                                                                        prepare, prepare, prepare the individual           and minimize their impact on                     his bike.
 can do to manage these difficulties and     Working memory, like processing              However, unlike Alzheimer’s Disease,
                                                                                                                                        for those changes in routine.                      daily functioning. ■
 optimize the functioning of the affected   speed, deals with the amount of              memory in patients with HD is often
 individual.                                                                             better than it appears. When given cues        The coping mechanisms mentioned above
                                                                                         or reminders, individuals with HD can          are things that a patient and his/her family
                                                                                         show that their memory can be quite            can do to deal with cognitive difficulties.
                                                                                         good. Examples of cues include making          There are also things that health care
                                                                                         lists for the grocery store or using           providers can do to help with these                           Centers of Excellence: Hot Topics
                                                                                         dry eraser boards to keep track of             symptoms of HD. Speech therapy, for
                                                                                         appointments. These cues jog the               example, can assist with communication                           • The George G. Powell HDSA              • Researchers at the HDSA Center
                                                                                                                                                                                                           Center of Excellence at North            of Excellence at the University of
                                                                                         memory of the HD patient, which is the         problems that can occur in the disease.
                                                                                                                                                                                                           Shore University Hospital is             Washington (WA) are looking for
                                                                                         little bit of extra help they need at times.   Physical therapy can be used to adapt                              seeking to develop state of the          biomarkers of HD that can be
                                                                                                                                        to motor skills deficits. Cognitive                                 art imaging methods for use as           used in prognosis, assessment
                                                                                         Other ways to improve memory
                                                                                                                                        rehabilitation can teach patients methods                          biomarkers in clinical trials of new     of severity and longitudinal
                                                                                         include: 1) using mnemonic strategies                                                                             therapies for movement disorders.        evaluations. One study is looking
                                                                                                                                        to compensate for their memory, language,
                                                                                         (e.g., HOMES is the first letter of each                                                                           Researchers have used Positron           at muscle tissue, one is looking at
                                                                                                                                        and problem solving difficulties.
                                                                                         of the Great Lakes); 2) pre-organizing                                                                            Emission Tomography and MRI to           spinal fluid and the third is looking
                                                                                         information that needs to be remembered                                                                           study disease progression both in        at MRI/SPECT of both brain and
                                                                                                                                        Although some medications for memory
                                                                                                                                                                                                           presymptomatic and symptomatic           muscle. A fourth study is evaluating
                                                                                         (e.g., write the grocery list in the order     problems in Alzheimer’s Disease (e.g.,                             HD. The goal of the research             the long term neuropathological
                                                                                         of the aisles in the store); and 3) limiting   donepezil) have been tried in HD,                                  project is to facilitate and hasten      and biochemical changes in people
                                                                                         information to what is necessary.              the results have not been particularly                             the screening and testing of new         with HD who had fetal brain tissue
                                                                                                                                        encouraging. Nonetheless, other                                    therapies for HD.                        transplants many years ago.

12 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                                Huntington’s Disease Society of America

                                                                                                                                                                                                                                                                         H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
              Risk                                                                                                                                   atAtrisk

                The Life Given Us
                                                                                                                                               was most thankful for his family. We
                                                                                                                                               felt heavy and sick with the burden of
                                                  By Nancy Hummer                                                                              what we now knew, and our minds were
                                                                                                                                               foremost on my brother’s children who
                                                                                                                                               might possibly face this disease, too.

                  hat happened to grandma?”                                                                                                    My analogy of our family situation,
                  I asked my father more than                                                                                                  although harsh, speaks to how I felt that
                  once through my life, as I                                                                                                   day: imagine a terrorist walks into your
    struggled with his insufficient answers.                                                                                                    home and informs you that he is going
    No minor question – I repeated whatever                                                                                                    to obliterate your family, by causing a
    I learned to my brothers, who came to                                                                                                      disease that will slowly disintegrate the
    me to speak in hushed tones about what                                                                                                     mind and body of half, or maybe more,
    this meant for our own future. The way I                                                                                                   of your family members. Then this
    puzzled it was this: how could a person,                                                                                                   terrorist asks, “Do you want to know who
    one of the first women to graduate in                                                                                                       I am going to pick? Or do you want to
                                                                                                                                                                                               often of the vision I had of myself as a          to look for and expect something new
    journalism at Ohio State, someone so                                                                                                       just wait and see?” What form of torture
                                                                                                                                                                                               wise old artist. What a rare gift to be           in its place, however bittersweet or
    active in the mind, so lively a dancer                                                                                                     could possibly be worse? This disease is a
                                                                                                                                                                                               given so clear an image about what I              discomforting. Here is what I think about
    with striking good looks, come to sink                                                                                                     terrorist, and this is the nature of the war.
                                                                                                                                                                                               want from life! Perhaps it is not the             life today: it will undress us, render us
    so deep into depression? Dad explained
                                                                                                                                               Today, I wait for test results from the         “being” that is so important, but rather          bare to the bone, no matter who we are,
    it to me by saying she never recovered
                                                                                                                                               Johns Hopkins Huntington’s Clinic. So           the awareness that enables us to live             but if we dare to live with this truth, we
    from significant deaths of close friends
                                                                                                                                               far, I am the only one in my family to go       toward a dream. They also tell you that           can live beyond ordinary boundaries.
    and family. Later, when asked about her
                                                                                                                                               through with the test. Although I initially     no matter what the outcome of the test,           And dream of imagined and unimagined
    loss of speech and rigid movements, he
                                                                                                                                               assumed we should all know whether              you must live your life. So, rather than          possibilities for a cure that will erase
    said that the doctors thought she probably
                                                                                                                                               we have the gene mutation, I have               dwell on what has been taken, better              this terrorizing threat to our families. ■
    suffered a series of small strokes.
                                                                                                                                               since changed my philosophy and fully
    So when dad began manifesting                                                                                                              embrace whatever choice my siblings
    grandma’s familiar jerky movements we                                                                                                      make. As I continue with my aggressive
    wondered anew what was in the family                                                                                                       fitness routine, I think of my dancing
                                                                                                                                                                                                          Centers of Excellence: Hot Topics
    genes. We researched neurological                                                                                                          grandmother, my athletic father and
                                                                                                                                                                                                             • The HDSA Center of Excellence            • Researchers at the HDSA Center
    disorders but nothing seemed to fit. It                                                                                                     brothers; I look at my own body.                                at Hennepin County Medical                 of Excellence at Rush University
    is hard to imagine now how we missed                                                                                                                                                                       Center (MN) analyzed the role              Medical Center are studying falls
    it, but the description of Huntington’s                                                                                                    “This disease is a terrorist,                                   of hospice in the care of patients         which are common in HD and
                                                                                                                                                                                                               with end-stage HD. This was                have severe consequences. They
    Disease was far too extreme to match         Now middle aged adults, we thought              as I grieved for my grandmother who           and this is the nature of war.”                                 a retrospective study of the               are studying clinical predictors
    our observations. Our attempts to get        we were prepared for any clinical               endured our judgment for giving up and                                                                        characteristics of patients in a           (motor, cognitive, functional state,
    dad to a doctor fell on deaf ears, as the    diagnosis. We could not have known the          failing to overcome her adversity.            Somehow, I am learning that happiness                           single facility at entry into hospice,     medications, etc.) that predispose
    patriarch of our family was not about        gravity of the shockwave that was about                                                       is an autonomous energy that alights                            and the role that hospice played           to falls in the hope that modification
    to submit to a doctor. Medication, he                                                        But worse was to come. We accepted            even in the darkest of times. I can still                       in their management. This study            of these predictors may help in
                                                 to hit us. One of the first clear images to
                                                                                                 the test confirming dad’s Huntington’s                                                                         was presented in September at the          fall prevention. This center is also
    insisted, would only hamper his golf         rise up in me was the realization that I                                                      celebrate what I have in the here and
                                                                                                                                                                                                               World Congress on HD. In a new             working on the development of care
    game. After years of family discussion,                                                      Disease from a family doctor who was          now. I can still honor my physical being,
                                                 might not live to be a wise old artist and                                                                                                                    study this center will look at the         techniques that will help caregivers
    oftentimes heated, he was finally stopped                                                     ill-equipped to counsel us and left with      no matter what hand I have been dealt.                          role of music therapy in improving         with troublesome behaviors and
                                                 I felt the first wave of loss in an immediate,
    by a police officer who rescinded his                                                         no more knowledge than we came with           This is how I know I am ready for the                           gait in people with HD.                    disabilities in HD. There is also
                                                 visceral way. My brothers and I were
                                                                                                 and a deep sense of frustration. Dad’s        results of my test.                                                                                        a study that is assessing the
    driving privileges until he was examined     so used to joking about how we were
                                                                                                 response was more comforting. Not a                                                                                                                      effectiveness and safety of
    by a doctor.                                 becoming like dad, I was programmed                                                                                                                                                                      psychotropic medications on motor
                                                                                                 man prone to emotion, he reassured us         One of the questions they ask before you
                                                 to assume whatever he had was coming                                                                                                                                                                     and behavioral function in HD.
                                                                                                 that he felt great, did not suffer pain and   are tested is, “what will you do differently
                                                 my way. Now, I felt a sense of shame                                                          if the test comes back positive?” I think

14 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                                      Hu n ti n g ton ’s Di sea se Soc iet y of Am eri ca

                                                                                                                                                                                                                                                                               H u nti ng to n ’s Di s ea s e So ci e ty of A me ri ca
                                                                                          [   FEATURE STORY     ]                                                                                                               [   FEATURE STORY                                             ]

 Hospice:                                                                                                           Tai Chi for Individuals with
 Living Each Day in Comfort and Dignity                                                                             Huntington’s Disease                                        By Nicola Briggs, Certified Tai Chi Instructor

                            “Hospice is a way of caring for the         shown that many patients live longer        Tai Chi is an ancient Chinese health        It also provides an avenue for success
                            dying.” You often hear this answer          because of the excellent nursing care       system that focuses on harmonizing          and a sense of achievement. With its
                            when asking for a definition of hospice.     and the focus on comfort. Hospice           the body’s vital life forces, or “Chi”,     focus of acceptance of one’s abilities,
                            But hospice is so much more than            does not take away choices regarding        to provide the benefits of relaxation,       as well as limitations, this healing
                            just physically caring for terminally-ill   future care. If the patient’s condition     increased mental and physical               art can help to greatly improve
                            patients. In fact, the focus of hospice     improves, or if the patient and family      awareness, and gentle conditioning          self-esteem.
                            is on living each day to the fullest in     so choose, the hospice patient can          for every part of the body. It is
                                                                                                                                                                Tai Chi exercises can be performed
                            comfort and dignity, at home or in          be discharged and readmitted at a           characterized by slow, graceful
                                                                                                                                                                standing or seated. Performing Tai
                            a home-like setting, surrounded by          later time.                                 movement, performed either
                                                                                                                                                                Chi exercises can be challenging and
                            those you love.                                                                         standing or seated.
                                                                        Because the Medicare Hospice                                                            the sense of achievement that follows
                            Hospice offers the services of nurses,      Benefit, Medicaid Hospice Benefit,            Research has shown that consistent          a class is significant. Taking on new
                            home health aides, social workers,          and most private insurers cover             Tai Chi practice can significantly           challenges stimulates a feeling of
                            chaplains, counselors and volunteers        hospice care, there are usually no          improve balance and coordination,           satisfaction and accomplishment
                            to the patient and their family.            charges for care. Most hospices will        which has increased its acceptance          that greatly improves the quality of
                            Hospice can relieve the stress on the       work with patients who do not have          within the health care community.           life. Tai Chi is a safe and productive
                            caregivers by providing daily help          insurance coverage to ensure that           It also increases blood, lymphatic,         way for each individual living with
                            with care, cooking, housekeeping            they receive the services they need.        and cerebral-spinal fluid circulation,       Huntington’s Disease to explore their
                            and even errands. Counseling and            Hospice pays for medical supplies,          which has a nourishing effect on the        personal power.
                            bereavement support for the patient,        equipment, and medications related          cells and all bodily systems. Principally
                                                                                                                                                                Performing Tai Chi exercises helps
                            their family and the circle of friends      to the terminal illness. Hospice also       used to improve the flow of energy
                                                                                                                                                                create a state of mental and physical
                            is also an integral part of the hospice     provides an on-call nurse, 24 hours         throughout the body, Tai Chi is an
                                                                                                                                                                relaxation. This relaxation can
                            holistic approach to care.                  a day, 7 days a week, giving the            effective form of self-healing that
                                                                                                                                                                temporarily reduce chorea and can
                                                                        caregiver the peace of mind in              gives students a concrete feeling of
                            One thing that sets hospice care apart                                                                                              be beneficial in delaying the onset of
                                                                        knowing that a nurse will respond           greater independence and confidence.
                            from other kinds of health care is that,                                                                                            muscular rigidity. It is a progressive
                                                                        if there is an emergency.                   This ancient practice allows students
                            whenever possible, the patient and                                                                                                  exercise, with each classroom
                                                                                                                    to assume greater responsibility for
                            family direct the care. Starting with       A comment heard over and over is,                                                       experience building upon the
                                                                                                                    their own mental and physical health,
                            admission and continuing with each          “We wish we had called hospice                                                          previous one, using repeated
                                                                                                                    which is the very essence of internal

   “                                                                                                                                                                                                              “
                            nursing visit, the needs and wishes of      sooner.” Hospice provides the                                                           movements to stimulate memory.
                                                                                                                    power. Tai Chi can be a very beneficial
        A comment heard     the patient and family are evaluated
                            and incorporated into an ever-changing
                                                                        greatest support when there is time
                                                                        to establish rapport with the patient
                                                                                                                    activity for people with HD.
                                                                                                                                                                It can reduce stress and improve
                                                                                                                                                                overall focus and concentration. Tai
                                                                                                                                                                                                                        Performing Tai
        over and over is,   and flexible plan of care. The ultimate      and the family – allowing months, not       Tai Chi exercises can increase range        Chi exercise helps to bring about an                    Chi exercises
                            goals of this process are self-determined   days. Hospice professionals cannot          of motion and muscle tone with their        acceptance of changes, while inspiring
        ‘We wish we had     life closure, aggressive pain and           take away the pain and sadness of           gentle stretching movements. Instructors    hope for the future. ■                                  helps create a
                            symptom control, and healthy grieving       saying goodbye to a loved one, but          focus on correct postural alignment
        called hospice      both for the patient and the family.        they truly do know how to help. You         during each class activity, which helps     Nicola Briggs is a Certified Tai Chi                     state of mental

                                                                                                                                                                Instructor who works with HD patients
        sooner.’            However, there are several things that
                                                                        can locate a hospice near you by            promote positive body awareness,
                                                                                                                                                                and others at the Terence Cardinal                      and physical

                                                                        contacting the National Hospice             both external and internal.                 Cooke Health Care Center in New York.
                            hospice does not do. Hospice does not
                            replace the caregiver and the family.
                                                                        and Palliative Care Organization,
                                                                                                                    Tai Chi practice is a positive way to
                                                                                                                                                                She can be reached at www.taichi                        relaxation.
                                                                        703-837-1500, or by going to their                                                      school.com/taichiforeveryone.html.
                            Hospice care does not hasten the dying                                                  manage and dissipate depression,
                                                                        website at www.nhpco.org. ■
                            process; in fact, recent studies have                                                   angry feelings and negative behavior.

16 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                              Huntington’s Disease Society of America

                                                                                                                                                                                                                                       H u nti ng ton’s Dis ea s e S oc iety of A m er ic a
      caregivers link
       Caregivers Link                                                                                                                       caregivers link
                                                                                                                                              Caregivers Link

             Caregivers as Advocates                                                                                                                Finding Facts on the Internet

 A                                                                                                                                      S
         family caregiver assumes            no background or experience in medical       Finding allies within the system is also             urfing the Internet for information      there are miracle cures that are being          When you find interesting research on
         the role of medical advocate,       settings or dealing with insurance           critical. Be on the lookout for a helpful            about health has become a common        withheld from the public. To find reliable       the Internet and want to share it with
         insurance manager and all           companies and it is easy to get frustrated   nurse, social worker or physician                    online activity. In fact, the Internet  information, it is wise to start your search    your doctor, try to narrow the information
 around ‘point person’ for their loved       and to lash out in anger. However, putting   who can give you an inside track to           has become a primary source of health          on the websites of reputable organizations:     down to the most important points you
 one. The uncertainty and concern of         a doctor, nurse or administrator on the      understanding how the system works.           information for many Americans. The            government agencies, not-for-profits and         wish to share and show the doctor that
 dealing with a loved one’s illness is       defensive will not improve the care of       The better you can communicate with           Pew Internet & American Life Project           entities known for their integrity. As an       you have garnered the information from
 aggravated by the complexities of our       your loved one. When confronted with         these individuals, the more guidance          recently reported that people with             example, Healthfinder.gov is a website           a reputable source. While more and
 healthcare system. Learning to navigate     a roadblock to care, remind yourself         they will be willing to give. The National    disabilities and                                             sponsored by the Office            more doctors are accepting their patients’
 successfully through the system takes a                                                  Family Caregiver’s Association has a series   chronic conditions                                           of Disease Prevention and         internet research and will gladly discuss
 combination of patience, perseverance
                                             “Becoming informed about                     of articles to give caregivers the tools      such as HD are some
                                                                                                                                                                      “... the Internet has Health Promotion for the                   what you have found, your doctor may
 and courage.                                your loved one’s illness is                  they need to be better communicators          of the most avid users        become a primary               U.S. Department of Health         not have time to look through a mass
                                                                                          and effective members of the healthcare       of Internet health sites                                     and Human Services. It is a       of website printouts.
 It takes patience to get the information
                                             also crucial to being an                     team. These articles can be found online      and that their online         source of health               solid starting point for finding
 and attention you need from a system                                                                                                                                                                                                  It can be encouraging to stay abreast
                                                                                          at www.thefamilycaregiver.org in the          searches affected                                            legitimate, up to date health
 that is not friendly to patients or         effective advocate.”                         Caregiving Resources section.                 treatment decisions,
                                                                                                                                                                      information....”               information from a variety
                                                                                                                                                                                                                                       of current research trends using the
 caregivers. It takes perseverance to                                                                                                                                                                                                  Internet. Scientists and investigators
                                             of what you want to accomplish in the                                                      their interactions                                           of sources. HDSA’s own
 politely and firmly demand that your                                                      Becoming informed about your loved                                                                                                           around the world are diligently striving
                                             short term. What are your immediate                                                        with their doctors, their ability to cope      website (www.hdsa.org) has links to
 questions be answered, and it takes                                                      one’s illness is also crucial to being an                                                                                                    toward breakthroughs in understanding
                                             goals and how can this person help you                                                     with their condition, even their diet and      resources, current research and a wide
 courage to trust that you have an                                                        effective advocate. The more you know,                                                                                                       and treating HD and its symptoms. HDSA
                                             accomplish them? A person who feels                                                        fitness routines. People are taking what        variety of publications you can download.
 important role to play as an advocate for                                                the more effectively you will be able to                                                                                                     and its partners, CHDI, Inc., and the
                                             under attack is unlikely to go that extra                                                  they find on the Internet very seriously.       Other good sources of information
 your loved one. Now, how do you do it?                                                   communicate with doctors, insurance                                                                                                          Huntington Study Group are working to
                                             mile for you or your loved one. Manage                                                                                                    are the National Institutes of Health
                                                                                          administrators and others. HDSA has           Unfortunately, when it comes to                                                                expedite the movement of basic research
 Learning to communicate effectively is      the situation at hand and at a later time                                                                                                 (www.nih.gov), the National Institute
                                                                                          resources and information which can           information on the Internet, it falls entirely                                                 into effective treatments for the disease
 perhaps the most important skill a family   you can write a letter of complaint or                                                                                                    of Neurological Disorders and Stroke
                                                                                          help you gain a better understanding          on the user to determine the value and                                                         through the HDSA Research Pipeline
 caregiver can cultivate. Most of us have    seek other redress.                                                                                                                       (www.ninds.nih.gov), the HD Lighthouse
                                                                                          of the disease. A wide range of helpful       truth of what they read. Anyone can                                                            for Drug Discovery, which you can
                                                                                                                                                                                       (www.hdlighthouse.org) and the
                                                                                          publications can be downloaded from           publish on the Internet and there is plenty                                                    read about starting on page 3 of
                                                                                                                                                                                       Huntington’s Disease Advocacy
                                                                                          our website, www.hdsa.org.                    of information that is untested, inaccurate,                                                   this magazine. ■
                                                                                                                                                                                       Center (www.hdac.org).
                                                                                                                                        even dangerous. Because there is such
                                                                                          Especially with a complex disorder
                                                                                                                                        a vast amount of information (and
                                                                                          such as HD, the family caregiver has
                                                                                                                                        mis-information) to be found online, it
                                                                                          an important role to play as advocate
                                                                                                                                        is extremely important to use safeguards
                                                                                          for their loved one. You know the patient
                                                                                                                                        when surfing the web.
                                                                                          better than anyone and you will be
                                                                                          the one who will bear the impact of           When you use a search engine, such as
                                                                                          decisions that are made for their care.       Yahoo or Google, to research a medical
                                                                                          Learning how to communicate within            topic, you may be confronted with a
                                                                                          the healthcare system, to be confident         list of thousands of websites, with no
                                                                                          and persistent in asking questions will       guidance on their quality or accuracy.
                                                                                          help you become an equal partner in           Some websites are commercial, designed
                                                                                          the healthcare team. ■                        to sell things. Some are filled with
                                                                                                                                        unsupported information and conjecture.
                                                                                                                                        Some websites want you to believe that

18 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                          Hu n ti n g ton ’s Di sea se Soc iet y of Am eri ca

                                                                                                                                                                                                                                                                   H u nti ng to n ’s Di s ea s e So ci e ty of A me ri ca
       progress report
        Progress Report                                                                                                                           advocacy

              HDSA Centers of Excellence                                                                                                                 HDSA is Awarded       Grassroots Technical
              Receive Grant from New York State                                                                                                          Advocacy Grant from National Health Council

 I                                                                                                                                          I
     n April, HDSA was awarded $50,000           through the NYS Department of Health.        Gottfried, Chair of the NYS Assembly              n June, HDSA was selected from                clinician, a social worker and a non-family    Congress that convenes in January
     as part of the New York State budget        Each Center of Excellence will receive       Committee on Health. Through their                among 15 voluntary health agencies            chapter member plus two staff.                 while also pursuing re-classification of
     to provide partial support for three        approximately $16,000 to support             efforts, and those of HDSA staff, HDSA            by the National Health Council to                                                            eligibility through the Social Security
                                                                                                                                                                                              In phase 2, our task team discussed
 HDSA Centers of Excellence located in           medical and social services offered to       was able to secure this partial funding       receive a year of consulting services                                                            Administration itself.
                                                                                                                                                                                              several issues that challenge our families
 New York at: the University of Rochester,       HD families in their region.                 for one year while also using the             from a Washington DC based advocacy
                                                                                                                                                                                              including those regarding long term care,      As the team moves into phase 4, it
 Columbia Health Sciences/NYS Psychiatric                                                     opportunity to develop a model that           firm. The purpose of the $25,000 “in
                                                 HDSA would like to thank volunteer                                                                                                           genetic discrimination, universal insurance    will be asking advocates who reside
 Institute and the George G. Powell Center                                                    may be used in other states to support        kind” grant is to build upon the advocacy
                                                 Katie Moser and HDSA Center of                                                                                                               coverage, mental health benefits, local         in key districts of House and Senate
 of Excellence at North Shore University                                                      the medical and social services provided      network HDSA has in place and to
                                                 Excellence director, Dr. Andrew Feigin,                                                                                                      lobbying to support HDSA Centers of            Subcommittees who govern Social
 Hospital. The award will be administered                                                     by centers in that state. ■                   expand the grassroots efforts of the agency
                                                 who met with Assemblyman Richard                                                                                                             Excellence (see accompanying piece on          Security to actively join its efforts to help
                                                                                                                                            in order to effect either a legislative or
                                                                                                                                                                                              page 20), Medicare eligibility and Social      introduce the legislation by meeting with
                                                                                                                                            policy change.
                                                                                                                                                                                              Security disability eligibility (SSDI). Task   their representative either in Washington,

 HDSA Recognizes                                            Our National Family Caregivers                                                  The grant consists of five phases:
                                                                                                                                            creating a working group of no more
                                                                                                                                                                                              team members unanimously chose to
                                                                                                                                                                                              lend their efforts to the re-classification
                                                                                                                                                                                                                                             DC or in their home district.

                                                                                                                                                                                                                                             Phase 5 will bring the implementation

                                                                                                                                            than 8 people comprised of 50%                    of disability eligibility for HD under
           ational Family Caregivers             • Remind caregivers to protect their         • Help a family caregiver decorate their                                                                                                       of the plan. As the team moves forward
                                                                                                                                            volunteers and 50% staff, identifying             social security and, as a sub-task, to
           Month is celebrated each                health, reach out for help, and speak        home for the holidays or offer to address                                                                                                    with this grant, the Washington-based
                                                                                                                                            the objective, developing a campaign,             work toward obtaining a waiver to the
           November to thank, support,             up for their rights.                         envelopes for their holiday cards.                                                                                                           advocacy consultant will be lending
                                                                                                                                            securing resources and implementing               two-year waiting period for Medicare
 educate and celebrate more than 50                                                                                                                                                                                                          his expertise at each stage of the plan
                                                 • Offer a few hours of respite time to a     • Find 12 different family photos and have    the campaign.                                     benefits once a person with HD is
 million family caregivers across the                                                                                                                                                                                                        to ensure that the team maximizes its
                                                   family caregiver so they may spend           a copy center create a 2008 calendar                                                          deemed eligible for SSDI.
 country who currently provide an estimated                                                                                                 In phase 1, HDSA sought volunteers                                                               efforts and utilizes the breadth of HDSA’s
                                                   time with friends or simply relax.           that the family caregiver can use to
 $306 billion in “free” caregiving services.                                                                                                who represented a broad share of our              Currently in phase 3, the task team            grassroots network. If you are not already
                                                                                                keep track of appointments and events.
                                                 • Send a card or a bouquet of flowers to                                                    stakeholders. Our task team consists              is developing a campaign that is two           an HDSA advocate, please register today
 HDSA is pleased to be an endorsing
                                                   brighten up a family caregiver’s day.      • Offer to prepare a holiday dinner for a     of 6 volunteers – a person who is at risk,        pronged – drafting a federal piece of          so we can keep you up to date with the
 organization and a part of the campaign
                                                                                                caregiving family in your community, so     one who is living with HD, a person who           legislation that will combine both             task team’s progress. Complete the form
 to bring attention to the needs of family       • Offer to help with home repairs or
                                                                                                they can just relax and enjoy the day. ■    is gene positive but not yet symptomatic, a       objectives (re-classification and the           below or go online to register today.
 caregivers. Here are some ways that you           indoor house cleaning or encourage a                                                                                                       waiver) for introduction in the new            Together, we can achieve this goal. ■
 can help a caregiver:                             local business to donate these services.

                                                                                                                                            Please help us update our records.
                              For more information visit: www.thefamilycaregiver.org or call 800-896-3650.

 Do you give at work?                            most respected charities, partner together
                                                 to offer workplace giving in companies
                                                                                              If your employer does not currently offer
                                                                                              a workplace giving campaign, and you
                                                                                                                                            If you are not currently a member of our emailing list, or if your
                                                                                                                                            email may have changed recently, please contact HDSA so you
                                                                                                                                                                                                                     Visit the Advocacy page of our website (www.hdsa.org then click
                                                                                                                                                                                                                     on Advocacy/Become an Advocate) to join or send an email to
                                                 across the country.                          would like to find out more about how          can be added to our e-list serve to receive late-breaking news,          Robert Coffey at coffeyr@hdsa.org with “update my contact info”
 The fall season is often when corporations
                                                                                              you can start one, please contact Alex        HD community updates, and advocacy action alerts.                        in the subject line.
 and the government conduct what is called       If your employer conducts a workplace
                                                                                              Roque at HDSA by calling 800-345-4372
 a workplace or employee giving campaign.        giving campaign and includes Community
                                                                                              ext. 233.
 This is when an employer makes it easy for      Health Charities as a partner, you can                                                     Name: ____________________________________________________ Address: ________________________________________
 its employees to dedicate dollars from their    make a difference in someone’s life today    HDSA has a new Community Health
 regular paycheck and donate them to the                                                                                                    City: ______________________________________________________ State: ________________ Zip: _____________________
                                                 by designating your contribution to HDSA.    Charities and Combined Federal Campaign
 charity or charities of their choice. Through   Federal employees can donate to HDSA         number! Please be sure to use 11238 to        Evening Phone: _____________________________________________ Email: __________________________________________
 Community Health Charities, HDSA, along         through the Combined Federal Campaign        designate contributions to the Huntington’s
 with a family of nearly 50 of America’s         by using the CFC code 11238.                 Disease Society of America.                   Are you currently receiving emails from HDSA? (circle one) [ yes ] [ no ]
                                                                                                                                            Please return this form to HDSA using the envelope provided in this magazine or email Robert Coffey at coffeyr@hdsa.org.

20 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                                  Huntington’s Disease Society of America

                                                                                                                                                                                                                                                                           H u nti ng to n’s D i sea se S o c i ety o f A m er i c a
      resource directory
        Resource Directory                                                                                                            resource directory
                                                                                                                                        Resource Directory

             A Guide to HDSA’s
                                                                                       MID-ATLANTIC REGION                      GREAT LAKES REGION                     Arkansas Affiliate*                         NORTHWEST REGION
                                                                                       (DE, PA, WVA, VA, MD, WASH DC)           (MI, IN, OH, KY, WI, MO)               Kansas Affiliate*                           (WA, OR, ID, MT)
                                                                                                                                (ST. LOUIS ONLY)                       Contact Janice Watsky

             Chapters and Affiliates
                                                                                       HDSA Chapters                                                                                                              Scotti Hutton
                                                                                                                                                                       HDSA Regional Development Director
                                                                                       Delaware Valley Affiliate*                Melissa Worthington                                                               HDSA National Development
                                                                                                                                                                       Tel: 504-712-0523 or
                                                                                       New Freedom Affiliate*                    HDSA Regional Development Director     800-558-3370                               Director West
                                                                                       Contact 610-260-0420                     Tel: 262-560-0494                      Email: jwatsky@hotmail.com                 Tel: 720-981-7577
 HDSA has placed professional staff in     NEW ENGLAND REGION                                                                   Email: mworthington@hdsa.org                                                      Email: shutton@hdsa.org
                                                                                       Delaware Valley Chapter                                                         Texas Affiliate
 strategic locations across the country    (CT, ME, MA, NH, RI, VT)                                                             HDSA Chapters                          www.hdsatexas.org                          HDSA Chapters
                                                                                       Tel: 610-260-0420 or
 to provide assistance in fund raising,    Contact Virginia Goolkasian                 (toll free) 877-384-3721                 Central Ohio Chapter                   HDSA Center of Excellence at Baylor        Northwest Chapter*
 awareness, education and care             HDSA Regional Development Director          www.dvhdsa.org                           Tel: 866-877-HDSA (4372) or            College of Medicine (see page 24)          Tel: 206-464-9598 or
                                           Tel: 978-454-8102 or                                                                 614-460-8800                                                                      888-264-4372
 initiatives offered by HDSA in                                                        Delaware Chapter                         www.hdsacentralohio.org
                                           (toll free) 888-554-8102                                                                                                    UPPER MIDWEST REGION                       Email: nwhdsa@yahoo.com
 support of HDSA’s mission.                                                            Tel: 877-384-3721                                                                                                          www.geocities.com/nwhdsa
                                           Email: virginia@hdsa-ne.org                                                          Indiana Chapter                        (ND, SD, NE, MN, IA, IL)
                                                                                       Maryland Chapter                         Tel: 317-271-0624 or                                                              HDSA Center of Excellence at University
 Regional Development Directors are        HDSA Chapters                               Tel: 800-416-4372                                                               Leigh Peterson
                                                                                                                                (toll free) 866-488-0008                                                          of Washington (see page 24)
 responsible for the states listed         Connecticut Affiliate*                                                                www.hdsaindiana.org                    HDSA Regional Development Director
                                                                                       Washington Metro Chapter
 including all chapters, affiliates and     Maine Affiliate*                             Tel: 703-204-4634 or                                                            Tel: 763-502-1407                          NORTHERN CALIFORNIA REGION
                                                                                                                                Kentucky Chapter                       Email: lpeterson@hdsa.org
 HDSA Centers of Excellence within         New Hampshire Affiliate*                     703-323-1403                                                                                                               (NO, CA, NV)
                                                                                                                                Tel: 502-637-4372 or
                                           Rhode Island Affiliate*                                                                                                      HDSA Chapters
 that region. Regional Development                                                     Western Pennsylvania Chapter             800-784-3721                                                                      Penny Riley
                                           Vermont Affiliate*                                                                    Email: hdsakychapter@bellsouth.net
 Directors work directly with HDSA                                                     Tel: 412-833-8180 or                                                            Illinois Chapter                           HDSA Regional Development Director
                                           Massachusetts Chapter*                      888-779-HDSA (4372)                      Michigan Chapter                       Tel: 630-443-9876                          Tel: 916-927-4400
 volunteers to maximize local resources    Contact Virginia Goolkasian                 www.hdsawpa.org                          Tel: 989-832-4170 or                   www.hdsa-il.com                            Email: priley@hdsa.org
 and efforts in the care and cure of HD.   HDSA Regional Development Director          HDSA Centers of Excellence at Johns      800-909-0073                           Iowa Chapter                               HDSA Chapters
                                           www.hdsa-ne.org                             Hopkins and The University of Virginia   http://hdsa-mi.com
 Information changes constantly. Please                                                                                                                                Tel: 866-248-IAHD (4243) or
                                           New England HDSA Center of Excellence       Health Sciences (see page 24)                                                   712-542-4976                               Northern California Chapter
                                                                                                                                Northeast Ohio Chapter
 contact HDSA at 800-345-HDSA for          (see page 24)                                                                                                                                                          Tel: 916-734-6277
                                                                                                                                Tel: 440-423-HDSA (4732)               Minnesota Chapter                          www.hdsanortherncalifornia.org
 updated information or go to the HDSA                                                 SOUTHEAST REGION
                                           GREATER NEW YORK REGION                                                              Ohio Valley Chapter                    Tel: 612-371-0904
 national website at www.hdsa.org                                                      (NC, SC, AL, GA, FL)                                                                                                       HDSA Center of Excellence at University
                                                                                                                                Tel: 513-741-HSDA (4372)               www.hdsa-mn.org
                                           (NY, NJ, FAIRFIELD COUNTY, CT)                                                                                                                                         of California, Davis Medical Center
 and click on “community” and then                                                     HDSA Chapters                                                                   Sioux Valley Chapter                       (see page 24)
                                                                                                                                St. Louis Chapter
 “HDSA near you.” ■                        Chenell Tannure                             North Carolina Chapter                                                          Tel: 605-334-9917
                                                                                                                                Tel: 314-961-4372 or
                                           HDSA Special Events Coordinator                                                      866-707-4372                                                                      SOUTHERN CALIFORNIA REGION
                                                                                       Contact Worth Bolton                                                            HDSA Centers of Excellence at Rush
                                           Tel: 212-242-1968 ext. 221                                                           Email: pcribbin@hdsa.org                                                          (SO. CA, S. NV)
                                                                                       www.hdsanc.org                                                                  University Medical Center (IL), Hennepin
                                           Email: ctannure@hdsa.org
 HDSA NATIONAL DEVELOPMENT                                                             Georgia Chapter                          Wisconsin Chapter                      County Medical Center (MN) and The
                                                                                                                                                                                                                  Lisa Gaetje
 MANAGER–EAST                              HDSA Chapters                                                                        Tel: 414-257-9499 or
                                                                                                                                                                       University of Iowa Hospitals and Clinics
                                                                                       Tel: 770-729-9207                                                                                                          HDSA Regional Development Director
                                                                                                                                                                       (see page 24)
                                           Long Island Affiliate*                       www.hdsa-ga.org                          877-330-2699                                                                      Tel: 866-920-0601
 Alex Roque
                                           New York Metro Affiliate*                                                             www.hdsawi.org                                                                    Email: lgaetje@hdsa.org
 HDSA National Office                                                                   Central Florida Affiliate                                                        ROCKY MOUNTAIN REGION
 Tel: 212-242-1968 ext. 233                Contact Chenell Tannure                                                              HDSA Centers of Excellence at Ohio
                                                                                       Contact Karen Milek                                                             (NM, UT, WY, AZ, CO)                       HDSA Chapters
 Email: aroque@hdsa.org                                                                                                         State University, Indiana University
                                           New Jersey Chapter                          Tel: 407-719-2291                                                                                                          Greater Los Angeles Chapter
                                                                                                                                and Washington University School       HDSA Chapters
                                           Tel: 609-448-3500                           www.huntingtonsorlando.org
                                                                                                                                of Medicine (see page 24)                                                         Tel: 888-4-HDSA LA or
 FIELD ADMINISTRATIVE                      Email: crokicki@hdsa.org                                                                                                    Arizona Affiliate
                                                                                       South Florida Chapter                                                                                                      800-686-9868
 COORDINATOR                               www.hdsanj.com                              Tel: 305-274-7411                                                               Tel: 888-267-3411 (AZ only)
                                                                                                                                SOUTHWEST REGION                                                                  Orange County Affiliate
 Megan Bonstein                                                                        www.huntingtonsdiseasefl.org                                                     Email: HDSA_AZ@hotmail.com
                                           Upstate New York Chapter                                                             (TX, OK, LA, MS, AR, KS, TN, MO)                                                  Contact Lisa Gaetje
 HDSA National Office                       Alex Roque                                  Alabama Support Group                    (EXCEPT ST. LOUIS)                     Rocky Mountain Chapter
                                                                                                                                                                                                                  HDSA Regional Development Director
 Tel: 212-242-1968 ext. 218                Tel: 212-242-1968 ext. 233                  Tel: 205-325-3877                                                               Tel: 303-321-5503 or
 Email: mbonstein@hdsa.org                                                                                                      Janice Watsky                          877-740-HDSA or 303-837-9937               Tel: 866-920-0601
                                           www.hdsauny.org                             HDSA Centers of Excellence at Emory                                                                                        Email: lgaetje@hdsa.org
                                                                                                                                HDSA Regional Development Director     www.hdsarockymountain.com
                                           HDSA Centers of Excellence at University    University, University of Alabama
 HDSA NATIONAL DEVELOPMENT                 of Rochester, Columbia Health Sciences/                                              Tel: 504-712-0523 or                   HDSA Center of Excellence at Colorado      San Diego Chapter
                                                                                       Birmingham and The University of
 DIRECTOR–WEST                                                                                                                  800-558-3370                           Neurological Institute (see page 24)       Tel: 800-473-4014 or
                                           NYS Psychiatric Institute and North Shore   South Florida (Tampa) (see page 24)
                                                                                                                                Email: jwatsky@hotmail.com                                                        760-752-1844
 Scotti Hutton                             University Hospital (see page 24)
 Tel: 720-981-7577                                                                                                              HDSA Chapters
                                                                                                                                                                                                                  HDSA Centers of Excellence at University
 Email: shutton@hdsa.org                                                                                                        Oklahoma Chapter*                                                                 of California Los Angeles and The University
                                                                                                                                Tel: 405-236-4372 or 877-232-4372                                                 of California San Diego (see page 24)
                                                                                                                                Email: okhdsa@sbcglobal.net

22 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                      Hu n ting ton ’s Disease S ociety of America

                                                                                                                                                                                                                                               H unt ing ton’s Disea se Soci ety o f Am erica
       resource directory
         Resource Directory                                                                                                                   researc h
                                                                                                                                                HDSA Research

              Centers of Excellence                                                                                                        Centers of Excellence: Hot Topics

                                                                                                                                              • The HDSA Center of Excellence at               • The HDSA Center of Excellence at the              specimens from people with HD. These
                                                                                                                                                the University of Iowa is working in             University of California Los Angeles is           revealed that the activity of complexes
 HDSA Center of Excellence program             NEW ENGLAND                                  SOUTH/SOUTHWEST REGION                              collaboration with the JHD working               conducting a pilot phase II double blind,         II, III, and IV of the mitochondrial electron
 serves as the cornerstone of HDSA’s           New England HDSA Center of Excellence        HDSA Center of Excellence at Baylor                 group of the European HD Network in              placebo controlled study of the safety            transport system is reduced in the
 commitment to care. These 21 facilities       Charlestown, MA                              College of Medicine, Houston, TX                    a project called JHD Registry/Natural            and tolerability of Curcumin C3 Complex           striatum thus suggesting that reduced
                                               Tel: 617-726-5532                            Tel: 713-798-3951                                   History Study to obtain data and vital           in patients with mild to moderate HD.             cerebral oxidative phosphorylation may
 provide a vast array of medical and
                                                                                                                                                information from JHD families in an effort       In a second study, they are looking at            be important in the pathogenesis of
 social services to HD families nationwide                                                                                                      to modify rating scales so they are more         whether there are EEG abnormalities               neuronal death.
                                               GREATER NEW YORK REGION                      MIDWEST REGION
 and work in collaboration with HDSA                                                                                                            suitable for monitoring the disease              in HD.
                                               HDSA Center of Excellence at the             HDSA Center of Excellence Washington
 chapters, affiliates, regions and support                                                                                                       progression in juvenile onset HD. They                                                            • The HDSA Center of Excellence at
                                               University of Rochester, Rochester, NY       University School of Medicine, St. Louis, MO
 groups to form a seamless national                                                                                                             are also working on a qualitative study        • The HDSA Center of Excellence                      Ohio State University is conducting
                                               Tel: 585-273-4147                            Tel: 314-362-3471
                                                                                                                                                (JHD Qualitative Study) that builds on           at Baylor College of Medicine                      a gait study. Individuals with HD are
 network of resources and referrals for        HDSA Center of Excellence at Columbia                                                            a similar study conducted in the UK to           is working on a study to determine                 commonly prescribed ambulatory
 those affected by HD and their families.      Health Sciences/NYS Psychiatric Institute,   UPPER MIDWEST REGION
                                                                                                                                                identify the needs of individuals and            whether tetrabenazine helps people                 assistive devices such as canes and
                                               New York, NY                                 HDSA Center of Excellence at Hennepin               families affected by JHD.                        with HD with balance, walking and hand             walkers to prevent falls. However, there
 HDSA Centers of Excellence also offer         Tel: 212-305-9172                            County Medical Center, Minneapolis, MN
                                                                                                                                                This Center is also conducting a clinical        function. This pilot study will provide            are no standardized clinical guidelines
 family members the opportunity to be a                                                     Tel: 612-873-2595 (new appointments)
                                               George G. Powell HDSA Center of                                                                  trial (CIT-HD) for individuals who are           preliminary data regarding this issue but          for prescription of such aides for people
 part of the search for effective treatments                                                612-873-2515 (appointments)
                                               Excellence at North Shore                                                                        not currently taking anti-depressant             it is also a test of several measurements          with HD and limited knowledge about
                                                                                            612-873-2522 (nurse line for est. patients)
 and a cure through clinical trials. As        University Hospital, Great Neck, NY                                                              medications, who notice mild changes             of function to see if they are a sensitive         the influence of the various devices
                                               Tel: 516-570-4477                            HDSA Center of Excellence at University                                                              measure and could be of use in future              on a person’s gait pattern. This
 research advances, there will be a                                                                                                             in their thinking ability and who are
                                                                                            of Iowa, Iowa City, IA                                                                               clinical trials.                                   study compares the biochemical
 greater need for individuals who want                                                                                                          diagnosed with HD. The trial evaluates
                                               MID-ATLANTIC REGION                          Tel: 319-353-4307                                                                                                                                       characteristics of gait in terms of
                                                                                                                                                Citalopram (or Celexa) effects on HD.
 to be a part of finding the answers. To                                                                                                                                                                                                             spatiotemporal gait measures (i.e.
                                                                                            HDSA Center of Excellence at Rush                   Contact the Center for more information.       • The HDSA Center of Excellence at the
 find out more about clinical trials in your    HDSA Center of Excellence at Johns Hopkins                                                                                                                                                           velocity, cadence, stride length, and
                                                                                            University Medical Center, Chicago, IL                                                               University of South Florida conducted a
                                               University/Hospital, Baltimore, MD                                                                                                                                                                   duration) and kinematic measures of
 area, contact your Center of Excellence.                                                   Tel: 312-563-2900                                 • Also at the HDSA Center of Excellence            study on predicting health and quality of
                                               Tel: 410-955-2398                                                                                                                                                                                    trunk sway while study participants
                                                                                                                                                at the University of Iowa a QOL in HD            life for family caregivers of persons with
 Each HDSA Center of Excellence receives       HDSA Center of Excellence at                 ROCKY MOUNTAIN REGION                                                                                HD. They found no direct relationship              with HD use four different types of
                                                                                                                                                survey project is being conducted to
 an annual grant of $50,000 from HDSA.         University of Virginia Health System                                                                                                              between stress and caregiver well being            canes and walkers. Contact the
                                                                                            HDSA Center of Excellence at Colorado               develop a scale of quality of life measures
                                               Charlottesville, VA                                                                                                                               and that the impact of stressors was               Center for more information.
 These grants are funded through the                                                        Neurological Institute, Englewood, CO               in order to determine the effectiveness
                                               Tel: 434-924-2665, Helpline: 866-290-4528                                                        and efficacy of treatments, particularly          moderated by appraisals and protective
 support patient and family service                                                         Tel: 303-768-4600
                                                                                                                                                from patient and caregiver perspectives.         factors. Seventeen family caregivers
 programs. These annual grants are             SOUTHEAST REGION                                                                                                                                  were interviewed and completed scales
                                                                                            NORTHWEST REGION                                    Surveys will be open to pre-symptomatic
 funded through special events organized       HDSA Center of Excellence at                                                                     individuals, symptomatic HD persons              to measure stressors, appraisals,
                                                                                            HDSA Center of Excellence at University
 by HDSA chapters, regions, volunteers         Emory University, Atlanta, GA                                                                    and caregivers.                                  protective factors and outcomes.
                                                                                            of Washington, Seattle, WA
 and friends who recognize the important       Tel: 404-728-6364
                                                                                            Tel: 206-598-4030                                   Eye Tracking is a study designed to
 role that these Centers of Excellence         HDSA Center of Excellence at University                                                                                                         • The HDSA Center of Excellence at
                                                                                                                                                utilize advanced eye-tracking methods
                                               of Alabama, Birmingham, AL                   NORTHERN CALIFORNIA REGION                                                                           Washington University School of
 play in providing help to our families.                                                                                                        to establish a biomarker for HD by
                                               Tel: 205-996-7865                                                                                                                                 Medicine (MO) conducted two unique
 To find out more about fund raising                                                         HDSA Center of Excellence at University             investigating subtle motor and cognitive
                                                                                                                                                                                                 studies that looked at mitochondrial
                                               HDSA Center of Excellence at                 of California Davis Medical Center                  eye tracking problems in people at risk
 events in your area, please visit                                                                                                                                                               electron transport system (ETS) activity
                                               University of South Florida, Tampa, FL       Sacramento, CA                                      for HD.
 the HDSA website and click                                                                                                                                                                      in people with HD. The first found no
                                               Tel: 813-974-6022                            Tel: 916-734-6278                                   Atomoxetine & HD is a clinical trial for         statistically significant differences in
 on “Community”.                                                                                                                                individuals who have concerns about              platelet citrate synthase, complex I or
                                               GREAT LAKES REGION                           SOUTHERN CALIFORNIA REGION                          attention and focus and are diagnosed            complex I/III though there was a highly
 Information changes constantly. Please
                                               HDSA Center of Excellence at Ohio State      HDSA Center of Excellence at University             with HD. The trial evaluates atomoxetine         statistically significant reduction in striatal
 visit www.hdsa.org and click on                                                                                                                (or Strattera) effects on HD symptoms.
                                               University, Columbus, OH                     of California, Los Angeles, CA                                                                       volume in participants with HD compared
 “Getting Help” and then “Centers of           Tel: 614-688-8672                            Tel: 310-794-1225                                   Please contact this Center of Excellence         with normal control participants. The
 Excellence“ to obtain the most current                                                                                                         for more information on this clinical trial.     second study examined postmortem
                                               HDSA Center of Excellence at Indiana         HDSA Center of Excellence at University
 contact information. ■                        University School of Medicine                of California, San Diego, CA
                                               Indianapolis, IN                             Tel: 858-622-5824
                                               Tel: 866-488-0008

24 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                                       Hu nti ng ton ’s Di sease Society of Am er i ca

                                                                                                                                                                                                                                                                                Hunti ngton’s Di sea se Society of A meri ca
       HDSA anniversary
      40th 40th Anniversary                                                                                                                             HDSA anniversary
                                                                                                                                                       40th 40th Anniversary
             Concert to Celebrate                                                                                                                              Eleventh Annual Dinner Celebrates
             Woody and Marjorie Guthrie Huge Success                                                                                                           Lives of Woody and Marjorie Guthrie

                                                                                                                                                                                                         his year marked the 40th           recipients of the Marjorie Guthrie
                                                                                                                                                                                                         anniversary of the passing of      Leadership Award for their concern for
                                                                                                                                                                                                         legendary folk singer Woody        the health and well being of those
                                                                                                                                                                                                  Guthrie and the founding of what has      affected by HD; Donald Barr who
                                                                                                                                                                                                  become the Huntington’s Disease           received the HDSA National Community
                                                                                                                                                                                                  Society of America. Friends, family and   Service Award in recognition of his
                                                                                                                                                                                                  patrons gathered in the Starlight Room    leadership and support of HDSA and
                                                                                                                                                                                                  at the Waldorf=Astoria on Thursday,       its at risk youth; and Joan Baez who
                                                                                                                                                                                                  October 18, for an evening of music,      received the Woody Guthrie Award for
                                                                                                                                                                                                  pageantry and fun. Several generations    her support of social and political causes
                                                                                                                                                                                                  of the Guthrie family came together to    around the world.
                                                                                                                                                      Mary White, Anna Canoni, Barbara Boyle,     celebrate the event that honors Woody
                                                                                                                                                                                                                                            Live auctions of limited edition prints
                                                                                                                                                 Laura Gaffney, Tony Lechich, Nora Guthrie and    and Marjorie Guthrie. From Oklahoma,
                                                                                                                                                                              Claudia Marshall.                                             donated by Tony Bennett and Mary Jo
               Grand Finale with Billy Bragg, Steve Earle, The Tangents, Stone Gossard and Brad,                   Tim Robbins with Liz Weber,                                                    HDSA welcomed Mary Jo Edgmon,
                                                                                                                                                                                                                                            Edgmon added excitement to the evening
                                                                  Tom Morello and Tim Robbins.                       HDSA Northwest Chapter.                                                      current matriarch of the family and
                                                                                                                                                                                                                                            and, with a silent auction, raised more
                                                                                                                                                                                                  “baby sister” of Woody, who attended

                                                                                                                                                                                                                                            than $16,000. Special thanks to John
       rom the welcoming words of host            the driving force behind the concert.            everyone back on stage for the big finale.                                                      with her niece Marie Nunley and grand
                                                                                                                                                                                                                                            Mellencamp, Jason Ritter, Marianna
       Tim Robbins to the penultimate             Liz thanked the artists and reminded             Tom Morello (Rage Against the Machine),                                                        nieces Karen Nunley and Le-Anna Davis.
                                                                                                                                                                                                                                            Palka, Mary Jo Edgmon, Howard Lederer,
       version of “This Land Is Your Land”        everyone that we were together for               explained to the audience that it was                                                          Also making their first appearances with
                                                                                                                                                                                                                                            Bob Pearson, Dr. Jim Gusella, and the
 that had the entire house singing and            two purposes – to raise funds and                to be an interactive, sing-along,                                                              their mothers, Anna (daughter of Nora)
                                                                                                                                                                                                                                            numerous sponsors and co-chairs that
 jumping to the beat, the First Annual            awareness for HD – and to have a good            dance-along, tribute to Woody, and                                                             and Sarah Lee (daughter of Arlo), were
                                                                                                                                                                                                                                            made the evening such a success. As a
 Celebration of Woody and Marjorie                time. Over the next three-and-a-half hours       led the assembled all-star band (featuring                                                     two great-granddaughters of Woody and
                                                                                                                                                                                                                                            result, more than $125,000 was added
 Guthrie to benefit HDSA was a                     both objectives were accomplished.               Stone Gossard (Pearl Jam), Billy Bragg,                                                        Marjorie – Kaylee and Sophia.
                                                                                                                                                                                                                                            to the Woody and Marjorie Guthrie
 rousing success.                                                                                  Steve Earle, Robbins, members of Brad
                                                  Nora Guthrie, Anna Canoni and                                                                                                                   Nora Guthrie and her daughter Anna        Research Fund which directly supports
                                                                                                   and the Tangents) in a passionate
 Nearly one thousand celebrants packed            members of the extended Guthrie                                                                                                                 served as Chairs of the event. Claudia    the HDSA pipeline for drug discovery.
                                                                                                   driving version of the Guthrie classic,       The Oklahoma Guthrie’s: Mary Jo Edgmon and
 Webster Hall, in New York City on                family were on hand to participate                                                                    Le-Anna Davis (seated) with Karen and     Marshall, host of City Folk Morning       Since the very first Guthrie Awards
                                                                                                   “This Land,” including all of the original
 Wednesday, October 17. Some were                 in the evening’s celebration.                                                                                      Marie Nunley (standing).     heard daily on WFUV radio, reprised       Dinner, which honored Drs. Anne Young
                                                                                                   verses, and a few improvised on the spot.
 there to support HDSA; others were                                                                                                                                                               her role as Mistress of Ceremonies. The   and Ira Shoulson and folk singer/activist
                                                  The Tangents provided the first upbeat
 there for the music, but all left with                                                            Given the success of the event, plans                                                          dinner honored individuals in the areas   Arlo Guthrie in 1997, more than $1.4
                                                  set. They were followed by Tim Robbins
 an appreciation of what Woody and                                                                 are underway for future concerts. ■                                                            of medicine, research, community          million has been raised in support of
                                                  and Friends, who interpreted several of
 Marjorie had contributed to the country                                                                                                                                                          service and entertainment.                HDSA funded research.
                                                  Woody’s chestnuts. Next up was Brad,
 and our cause; and an appreciation for
                                                  in their first public performance in three        “...all left with an                                                                           We congratulate the James F. White HD     Now more than ever, the answers are
 the way in which the HD community
                                                  years. Steve Earle and Billy Bragg each                                                                                                         Unit at Terence Cardinal Cooke            within reach. Please consider making
 can pull together to raise funds
                                                  contributed their own rousing sets, full
                                                                                                   appreciation of what                                                                           Healthcare Center on receiving the        your donation today to support HDSA
 and spirits.
                                                  of Guthrie and Guthrie-esque tunes,              Woody and Marjorie                                                                             Guthrie Family Humanitarian Award for     and the pipeline for drug discovery. ■
 Tim Robbins set the tone for the evening         which had the crowd begging for more.                                                                                                           their dedication and compassionate care
 when he introduced Liz Weber, President                                                           had contributed to the                                                                         of people with HD and their families;
                                                  After auctioning off a guitar signed by
 of the Northwest Chapter of HDSA, and                                                                                                                                                            Alexis Borisy and CombinatoRx,
                                                  all of the performers, Robbins invited           country and our cause...”                       Don Barr with Barbara Boyle, Nora Guthrie,
                                                                                                                                                          Anna Canoni, Claudia Marshall and
                                                                                                                                                                        members of the NYA.

26 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                               Hu n ti n g ton ’s Di sea se Soc iet y of Am eri ca

                                                                                                                                                                                                                                                                        H u nti ng to n ’s Di s ea s e So ci e ty of A me ri ca
      ways to give
       Ways to Give                                                                                                                       ways to give
                                                                                                                                           Ways to Give

              There are many ways                                                                                                   There are many ways for you to
                                                                                                                                    make your contribution to HDSA:
                                                                                                                                                                                    Appreciated Stocks and/or Mutual
                                                                                                                                                                                    Funds – earn a charitable tax deduction
                                                                                                                                                                                                                                    Use your
              for you to make your contribution to HDSA                                                                             Please visit our website, www.hdsa.org,
                                                                                                                                                                                    for the full fair market value of the gift

                                                                                                                                                                                                                                    IRA in the
                                                                                                                                                                                    while you lower your capital gains taxes.
                                                                                                                                    and click on “To Make a Donation to HDSA
                                                                                                                                    Click Here!” This will take you to a secure     Life Insurance – the donation of a life

 H                                                                                                                                                                                                                                  fight to
           DSA is an organization that       complements work being performed            • HDSA Chapters and Affiliates work         page where you can make a direct dona-          insurance policy will earn you an income
           strives to make every dollar      by the HDSA Coalition for the Cure.           in tandem with HDSA Centers of           tion to HDSA. If you prefer, you can print      tax deduction equal to the lesser of the cash
           count. Your generosity has
 allowed us to develop programs that
                                           • CHDI, our partner in the translational
                                             phase of research, investigates how
                                                                                           Excellence to provide access and
                                                                                           information about community-based
                                                                                                                                    out the gift form and mail it to: HDSA,
                                                                                                                                    505 Eighth Avenue, Suite 902, New York,
                                                                                                                                                                                    value of the policy or total premiums paid.

                                                                                                                                                                                    Real Estate – a gift of a property that is
                                                                                                                                                                                                                                    end HD.
 positively impact the lives of people                                                     services and support.                    NY 10018, or fax it to 212-216-9004.
                                             basic research and early translational/                                                                                                fully paid off will allow you to remove
 with HD. With your continued support                                                                                                                                                                                               Using your IRA in planned giving
                                             preclinical research findings can be         • HDSA Social Workers work with            You may choose to establish a Family            that asset from your taxable estate. You
 we will expand these programs to help                                                                                                                                                                                              may have advantages for your
                                             applied to developing substances that         HDSA Chapters and HDSA Centers           Fund to honor your family or to                 can then deduct the fair market value
 the ever-increasing population of                                                                                                                                                                                                  heirs as well as your charity of
                                             will slow the progress or prevent HD.         of Excellence to ensure that HD          remember a loved one. Join with friends         of your gift while avoiding all capital
 people with HD.                                                                                                                                                                                                                    choice. That is because funds
                                                                                           families receive services to which       and relatives, and pool your resources,         gains taxes on the property. You can
                                           • CHDI and the Huntington Study                                                                                                                                                          from an IRA can be designated for
 HDSA puts your donations directly to                                                      they are entitled.                       to make your donated dollars work               transfer the deed of your home or farm
                                             Group are our partners that carry out                                                                                                                                                  a charitable organization without
 work, fulfilling the three objectives of                                                                                            harder than you could individually.             to HDSA now and retain the right to use
                                             clinical trials of potential treatments     • HDSA Support Groups provide the                                                                                                          triggering income, estate or
 our mission statement:                                                                                                                                                             the property for your lifetime and that of
                                             in humans.                                    HD community with critical emotional     Other options include an HDSA                                                                   transfer taxes. Using your IRA for
                                                                                                                                                                                    your spouse.
                                                                                           support as well as serve as an outlet    Tribute Gift: honor a friend or relative;                                                       charitable purposes can be a tax
 To find a cure for HD
                                           To care for and help people                     for resources and referrals.             or an HDSA Memorial Gift: honor the             For more information on the donation            smart bequest – freeing you to
 by funding:
                                           with HD and their families by                                                            memory of a loved one.                          of these financial instruments or programs,      give less heavily taxed assets, such
 • HDSA Coalition for the Cure             creating and funding:                         To educate the public and                                                                  please contact Leah Donnelly at the             as cash, securities and real estate,
                                                                                                                                    Your employer or organization may be
   investigators, who focus on basic                                                     healthcare professionals                                                                   HDSA National Office, 800-345-HDSA               to your family members.
                                           • HDSA Centers of Excellence – 21                                                        part of the HDSA Matching Gifts Program,
   and preclinical research to identify                                                  about HD by developing                                                                     (4372) ext. 217.
                                                                                                                                                                                                                                    In addition, under the Pension
                                             specialty clinics at major medical                                                     which doubles your donation. A list of
   and understand the bio-mechanisms                                                     and distributing:                                                                                                                          Protection Act of 2006, you can
                                             facilities located throughout the                                                      participants is available on our website.       Please visit our website regularly and
   that cause HD.
                                             United States where people with             • Materials that create awareness          If your employer is not part of this program,   browse the HDSA Marketplace.                    even use your IRA to make
 • HDSA Grants and Fellows are the           HD and their families receive quality         about HD for both the public and         we would be happy to help enroll your           Purchasing our Care2Cure Bracelets,             charitable gifts during your lifetime,
   next generation of HD investigators.      medical care, social services and             government sectors.                      company or organization. It’s another           40th Anniversary Bracelets, holiday             without negative tax effects. Under
   Research funded through this program      support. HDSA Centers of Excellence                                                    way for you to help HDSA, now, and              cards, amaryllis plants and other               the act, you can transfer up to
                                                                                         • Informational publications for people
   provides new insights into HD and         are also focal points for clinical trials                                              in the future.                                  merchandise makes a difference –                $100,000 directly to a charitable
                                                                                           with HD and their families.
                                             and genetic testing programs.                                                                                                          and helps us build awareness at the             organization such as HDSA. There
                                                                                                                                    In addition to these cash gifts, you can                                                        are two requirements, however:
                                                                                         • Professional publications and seminars                                                   same time.
                                                                                                                                    participate in our Planned Giving Program                                                       you must be 70 years of age or
                                                                                           for the healthcare community.
                                                                                                                                    that includes the HDSA Heritage Club            You can also make a valuable donation
                                                                                                                                                                                                                                    older and you must complete the
                                                                                         • Regional and national seminars,          for persons remembering HDSA in their           by offering your time, your skills, your
                                                                                                                                                                                                                                    transfer before December 31, 2007.
                                                                                           conferences and conventions for HD       wills; life income Charitable Remainder         knowledge to a local HDSA chapter. In
                                                                                           families and medical professionals.      and Annuity Trusts; and Charitable              the past year, volunteers have helped           Consider designating funds from
                                                                                                                                    Bequests. Other financial instruments            upgrade chapter websites, videotaped            your IRA for the fight against HD.
                                                                                         • National website (www.hdsa.org)
                                                                                                                                    that can be used for donations are:             Center of Excellence patients and provided      Through careful planning, you
                                                                                           that provides information to the world
                                                                                                                                                                                    other support services to chapters and          can make a generous gift while
                                                                                           wide HD lay and professional medical     IRA – funds from an IRA can be                                                                  ensuring that your heirs receive
                                                                                                                                                                                    affiliates, in addition to helping organize
                                                                                           community about new advances in the      designated to HDSA with no negative
                                                                                                                                                                                    events and participating in HDSA activities.    assets with the least tax exposure.
                                                                                           care and cure of Huntington’s Disease.   tax effects. If you are 70 ½ years of age                                                       Be sure to get expert advice when
                                                                                                                                    or older, you can transfer up to $100,000       The Cure is within reach. Please help us
                                                                                         • Referral services for people with HD                                                                                                     contemplating your options. ■
                                                                                                                                    to HDSA prior to December 31, 2007              reach this goal, and continue to make a
                                                                                           and their families.
                                                                                                                                    to receive the tax credit this year.            difference in the lives of every person
                                                                                                                                    Please see sidebar for more details.            with HD. ■

28 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                     Hu n ti n g ton ’s Di sea se Soc iet y of Am eri ca

                                                                                                                                                                                                                                                              H u nti ng to n ’s Di s ea s e So ci e ty of A me ri ca
      ways to give
       Ways to Give

             Welcome to the HDSA Marketplace                                                                                                                                           Kim Quaid (HDSA Center of
                                                                                                                                       World Congress...                                                                            as there is a probability that results can
                                                                                                                                       —continued from page 11.                        Excellence at Indiana University)            vary +1 or -1 in the CAG repeat length
                                                                                                                                                                                       discussed results from the PHAROS            and this variance can have a significant
                                                                                                                                       Andrea Ciarmiello (Italy) presented             observational study on factors associated    impact on individuals who have been
                                                                                                                                       a study which demonstrated the loss of          with pursuing genetic testing. Over 90%      given results of CAG repeats of 36-39.
                                                                                                                                       grey and white brain matter with disease        of individuals enrolled in PHAROS
 There are so many new ways that             Guthrie. Purchase your tee shirt today for    Don’t forget to order your “Winter          progression and a loss of grey matter in                                                     Douglas Langbehn (University of
                                                                                                                                                                                       chose not to be tested during the study,
 you can support HDSA every day.             just $25 plus shipping and handling.          Cabin” Holiday Cards: [E] and send          individuals who are gene positive and                                                        Iowa) presented data to validate CAG-
                                                                                                                                                                                       stating medical and societal concerns as
                                                                                           your message of support and good wishes     asymptomatic. Spinal fluid also showed                                                        based age of onset using data from the
 Items change constantly so check            Woody and Marjorie Guthrie                                                                                                                barriers to testing.
                                                                                           to your friends, family and business        an increase in white matter and a                                                            PREDICT observational study. A useful
 the Marketplace often. All proceeds         Concert Poster: [B] Own a limited
                                                                                           associates this season. Each box is $35     decrease in grey matter.                        Yvonne Bombard (Canada) found that           model was derived that expresses the
 benefit HDSA’s research, care and            edition poster of this incredible concert
                                                                                           (includes shipping and handling) and                                                        93 out of 167 individuals participating in   relationship between an individual’s
 education programs. If you have             that took place in New York’s famed                                                       Nicola Hobbs (UK) talked about
                                                                                           HDSA receives 40% of the price of your                                                      her study reported genetic discrimination    CAG repeat length and the probability
                                             Webster Hall for just $20 plus shipping                                                   whether an increased caudate atrophy
 any questions, please contact                                                             cards to benefit our programs. Visit our                                                     in reference to life insurance, disability   that the disease will manifest by a
                                             and handling.                                                                             rate could serve as a biomarker in early
 Mynelly Perez at ext. 214 or                                                              Marketplace Holiday Store today and                                                         insurance, making reproductive decisions     certain age.
                                                                                                                                       HD. Researchers used the caudate                and relationships. Genetic testing did
 email mperez@hdsa.org.                                                                    place your order to guarantee delivery
                                             Holiday Items – New for 2007                                                              boundary shift integral (CBSI) and                                                           Lucienne van der Meer (Netherlands)
                                                                                           in time for the holidays.                                                                   not increase the risk of discrimination
                                                                                                                                       found a significant difference between                                                        presented a study that found a correlation
 Ways to Show Your Support of                HDSA 40th Anniversary Awareness                                                                                                           as much as knowing family history at
                                                                                                                                       pre-manifest and early HD.                                                                   between aspects of personality,
 HDSA Every Day                              Bracelet: [C] This exclusive bracelet         Other Services                                                                              an early age.
                                                                                                                                                                                                                                    psychological and social characteristics
                                             was designed to celebrate HDSA 40th                                                       A. Weindt (Germany) posed that
 Care2Cure Bracelet: Our blue                                                              Flowers: FlowerPetal, an international                                                      Claudia Licklederer (Germany) spoke          and early symptoms of HD, with certain
                                             anniversary. The four large Swarovski                                                     striatal atrophy in HD is leftward bias.
 Care2Cure bracelet is great for walks,                                                    web florist, can help you solve your                                                         about the long term effects of predictive    personality types more likely to exhibit
                                             crystals represent each decade that we                                                    He looked at handedness in men and
 hoops or any event where people gather                                                    gift needs with fresh flowers, gift sets                                                     testing on mental health and quality of      cognitive symptoms and others to
                                             have fought to better the lives of those                                                  women and found a greater decrease in
 to support HDSA. Bracelets are $2 each                                                    and more. HDSA receives 12% of your                                                         life, as individuals with HD have a lower    express behavioral symptoms.
                                             affected by HD. The bracelet is accented                                                  grey matter on the left side of the striatum.
 and sell in lots of 10 (10/$20).                                                          purchase and, as a supporter of HDSA,                                                       quality of life and more depression than
                                             in Swarovski pearls and sterling silver.                                                  All participants were right handed.                                                          Sheila Simpson (UK) spoke about a
                                                                                           there is no charge for flower delivery                                                       control or gene positive participants,
 HDSA Circle of Hope Necklace:               Each bracelet is $40.00 and 100% of                                                                                                                                                    project that has been launched by the
                                                                                           (a $10 savings). Please visit FlowerPetal   Wim Vandenberghe (Belgium) studied              mainly due to a lack of social networks.
 Share your message of hope with this        the net profits benefit HDSA programs.                                                                                                                                                   Euro HD network to develop a standard
                                                                                           today at http://hdsa.flowerpetal.com.        cannabinoid type 1 receptor (CB1),
 stylish necklace that features the words                                                                                                                                              Reinier Timman (Netherlands)                 set of guidelines for care for those
                                                                                                                                       which is needed to signal behavior,
 “HDSA Circle of Hope” on one side and       Old Favorites                                 FTD: When you purchase through FTD                                                          presented a spreadsheet created to aid       affected by HD.
                                                                                                                                       maintain weight and moderate other              genetic counselors in assessing risk
 “Cure Huntington’s Disease” on the other.                                                 HDSA will receive a 15% donation at
                                             Amaryllis: [D] Each amaryllis comes                                                       functions, as a potential biomarker for                                                      Martha Nance (HDSA Center of
 Wear it on either side. The necklace is                                                   the end of the year. To order flowers                                                        for HD without a genetic test, using
                                             packaged to grow. Just unwrap, water                                                      HD, but found no useful correlations.                                                        Excellence at Hennepin County Medical
 available for $7.50 each or 2 for $10.                                                    or other FTD products, please go to                                                         current age, length of CAG repeat in
                                             and watch. This year, there are five colors.                                                                                                                                            Center) presented on end of life care
                                                                                           www.ftd.com/hdsa or call 1-800-SEND         Robert Wolf (Germany) studied working           close relatives, information on age at
 Woody and Marjorie Guthrie                  At $10 each, buy one in every color!                                                                                                                                                   and hospice, looking at HD patients who
                                                                                           FTD and mention code #3015 when             memory dysfunction in pre-symptomatic           onset, or pre-symptomatic or prenatal
 Concert Tee Shirt: [A] Be a part of         Purchase your amaryllis kits online at the                                                                                                                                             had died in one nursing facility between
                                                                                           purchasing. ■                               HD using reaction time. There was no            test results of their tested children and
 HDSA’s 40th Anniversary inaugural           Marketplace (deadline December 15) or                                                                                                                                                  2000 and 2006. The data suggests that
                                                                                                                                       difference between control participants         other information.
 concert in honor of Woody and Marjorie      from your HDSA chapter and affiliate.                                                                                                                                                   a large proportion of HD patients have
                                                                                                                                       and presymptomatic individuals, although        Carsten Saft (Germany) presented case        distressing terminal symptoms which
                                                                                                                                       there was evidence of a significant              data on the influence of modifier genes        may benefit from palliative care.
                                                                                                                                       decrease in the left dorsolateral prefrontal    with environmental factors that could
                                                                                                                                       cortex with the gene present.                                                                Sarah Tabrizi (UK) examined changes
                                                                                                                                                                                       result in HD manifestation with 34 CAG
                                                                                                                                                                                                                                    in those with pre and early HD over a
                                                                                                                                       Matthieu Robert (UK) presented a study          repeats. The case was confirmed with a
                                                                                                                                                                                                                                    two year period, using measures that
                                                                                                                                       of eye movements in pre-symptomatic             CCT scan.
                                                                                                                                                                                                                                    included imaging, eye movement, quality
                                                                                                                               E       people which may serve as a functional          Oliver Quarrell (UK) spoke about             of life, motor and cognitive assessments,
                                                       C                                                                               biomarker of HD.                                the need for quality control and             wet markers and neuropsychiatric
                                                                                                                                                                                       standardization across testing facilities,
                                                                                                               D                                                                                                                    —continued on page 32.

30 WINTER 2007 | HDSA.ORG                                                                                                                                                                                                                                        H u nt i ng t o n’s D i s e a s e S o c i e t y o f A me r i c a

                                                                                                                                                                                                                                                                 H u n t i n g t o n ’s D i s e a s e S o c i e t y o f A m e r i c a
 5th Annual Phyllis E. Dake...                model to test the compound on, and            The research supported by HDSA
 —continued from page 5.                      determine dosing and timing before            and presented at the Dake Research
                                              clinical trials on humans can begin.          Symposium is expanding our
 familiar compounds, such as creatine
                                              Dr. Hersch is working in developing           understanding of the basic processes
 and coenzyme Q10, which may have
                                              new biomarkers that can indicate if a         of HD. Your continued support for the
 general effects on energy and oxidation,
                                              drug is working. For example, brain size/     work of the HDSA Coalition for the
 and focusing on increasing their
                                              shrinkage has been identified as a             Cure is vital to the scientific selection
 specificity and potency. Novel
                                              biomarker that can be assessed using          of targets for therapies that underlies
 compounds identified by assay are also
                                              non-invasive imaging techniques. The          HDSA’s entire drug discovery pipeline. ■
 being investigated, but this is an arduous
                                              ability to use this as a biomarker shaved
 process. The researcher must determine
                                              5-6 years off the time required for
 whether the activities altered by the
                                              creatine to move forward in
 compound are a big piece of HD
                                              clinical trials.
 pathology, choose the correct mouse

 World Congress...                            results indicated that motor function and     Ruth Walker (Mount Sinai School of
 —continued from page 31.                     gait improved while chorea decreased          Medicine) gave a video presentation on
                                              with almost no adverse side effects, with     the disorders HDL2, McLeod Syndrome
 assessments. These will be used to assess    a strong trend towards improvement in         and chorea-acanthocytosis that mimic
 the effects of drugs in these groups.        symptoms of anxiety and depression.           HD and can complicate diagnosis.
 David Crauford (UK) gave a                   Diana Rosas (Massachusetts General            D.D. Rudnick (Johns Hopkins) gave
 presentation on treating sleeplessness,      Hospital) presented data on a high-dose       a short presentation on HDL2 and
 irritability and apathy and concluded        creatine study of 10 individuals. As the      discussed how a better understanding
 that there is no evidence to determine       dosage increased, the benefits of the          of the potential points of convergence
 which are the most effective drugs for       treatment decreased. After several weeks,     between HDL2 and HD could help to
 persons with HD.                             it was determined that 30 gr. of creatine     direct targets for therapies.
 Leigh Beglinger (University of Iowa)         was the optimal dosage to reduce weight
                                                                                            Hans Jung (Switzerland) discussed
 gave a presentation on the use of            loss, slow loss of motor skills, reduce the
                                                                                            another disease that mimics HD –
 Atomoxtine to enhance executive              formation of aggregates in the brain and
                                                                                            McLeod’s Syndrome. The study concluded
 function and attention in early HD.          reduce oxidative stress.
                                                                                            that while McLeod’s Syndrome is a
 This ongoing study began in May ‘07          Kevin Biglan (University of Rochester)        progressive multi-system disorder similar
 and will conclude in mid 2008.               presented a study proposing the               to HD, the brain pathology is not
 Ray Dorsey (University of Rochester)         examination of the effectiveness of three     similar to HD.
 presented the results of the Miraxion        dosages of CoQ10 (600, 1200 and 2400
                                                                                            Events such as the World Congress
 study (ethyl EPA) in both North America      mg./day) in pre-manifest individuals in
                                                                                            showcase the benefits of collaboration
 and Europe. Though the initial results       order to establish the optimal dose that
                                                                                            and the sharing of information as keys
 showed that the drug was safe and            could be used to prevent onset.
                                                                                            to advancing therapies. The breadth of
 well tolerated, there was no statistical     Adrienne Curtis (UK) reported on              clinical investigation presented at the
 significance at 6-months and the twelve       the use of Nabilone in a pilot study.         Congress is an indication of the HD
 month data has not yet been released.        Over a two year period, this synthetic        community’s dedication to rapidly
 Joakim Tedroff (Sweden) gave a               cannabanoid was shown to reduce               developing therapies to treat the
 presentation on the use of ACR16, a new      movements and improve mood. A                 symptoms of the disease and to
 dopamine stabilizing drug. In a phase 2      larger scale trial may be scheduled.          ultimately find a cure. ■
 open label study in Scandinavia, the


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