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					                                h d s a . o r g

                                                                                    A P R I L     2 0 0 8


                 Great Events, Great People and
                   Great Hope for the Future
                          By Barbara T. Boyle, National Executive Director/CEO
    have just returned from an amazing experience,Fahey families for all of their hard work over the

 I  one that re-energized me, and reminded me what
    people can do when they truly work together.
         The 8th Annual ADE for HD Dinner was held
                                                  last eight years. Joe Fahey, a long-time local HD
                                                  leader, received a special plaque in recognition of all
                                                  he has done for the organization over the years.
                                                  This event, held in a rural area with a population of
 on Saturday, April 19th at the Knights of Columbus
 Hall in New Prague, MN. There were 300 tickets   under 5,000, has raised over $200,000 since its
 sold with over 250 people in attendance - a full inception eight years ago.
 house! The evening started with a social, followed       And while the final accounting is underway
 by a great dinner and silent and live auction.   as I write this note, it looks as if this year’s event
         It was my great pleasure to be able to   raised over $30,000!
 speak to all who attended about how important            The ADE for HD Dinner is an amazing
                                                  example of grass roots fundraising. So often we
 their support is to our scientists and our Centers
 of Excellence, in addition to our educational andthink the best fundraisers come of out of the large
 advocacy efforts.                                metropolitan communities. But the reality is that in
         It was a particular honor for me to to besmall towns like Belle Plaine, MN, residents know
 able to give special recognition to the Stier andthe HD families, work along side them, go to
                                                                        church with them, send their

      Barbara Boyle and Leon Tibben flank honoree, Joe
                                                                        kids to school together, and on

      Fahey, at the ADE for HD Dinner in Belle Plaine, MN
                                                                        and on. When a member or
                                                                        family of a small community
                                                                        suffers from such a devastating
                                                                        illness such as HD, the turnout
                                                                        in support of that family can
                                                                        sometimes be phenomenal. This
                                                                        dinner is a perfect example of
                                                                        that phenomena, one I’ve seen
                                                                        in communities of all sizes
                                                                        throughout the U.S.
                                                                                   That’s really the key
                                                                        factor in our quest to find the
                                                                        answers to Huntington’s
                                                                        Disease: all of us working
                                                                        together, supporting each other,
                                                                        stepping in where help is needed
                                                                        (continued on page 4)



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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


              HDSA’s 23rd Annual National Convention
         June 6-8, Omni William Penn Hotel, Pittsburgh, PA
       he 23rd Annual HDSA National                                                 HDSA national Board members.
   T   Convention is just around the
   corner— if you haven’t made your
                                                                                      s
                                                                                    As k t h e S o c i a l S e c u ri t y
                                                                                    A d m i n i s t ra t i o n – On Friday and
   reservations as yet please do so today.                                          Saturday register to meet with rep-
   You can either fax in the registration                                           resentatives of the Social Security
   form found on pages 12 and 13 of this                                            Administration to discuss disability
   e-newsletter, or visit the HDSA website,                                         (SSDI), supplemental income (SSI)
   www.hdsa.org to register on-line. You’ll                                         and any other hot topic. You can
   find a complete program for this year’s                                          register for an appointment at the
   Convention on pages 14-20 of this e-                                             Social Security desk in the Exhibit
   newsletter.                                                                      Hall. Please note that information
             This year the HD Activity Center                                       provided will be for informational
   at convention will feature sessions in t’ai                                      purposes only.
   chi, yoga, music therapy, speech, and                                            A d vo c a c y – join HDSA for one of
   OT. Sessions are open to adults with HD                                          two advocacy workshops (new to
   and youth affected by JHD. The HD                                                advocacy and an advanced session).
   Activity Center will be open from 1:00                                           Learn about how HDSA is working
   – 5:30 p.m. Friday and Saturday. The center will              to revise the guidelines used by the Social Security
   be supervised by a nurse educator and volunteers              Administration to determine eligibility for disability.
   familiar with HD.                                             HDSA would like to thank the convention program
             New for the 2008 convention:                        committee for their work in preparing this year’s
   Op e n i n g Ce re m o n y – join us as we welcome the        program: Bruce Jennings, Chair; Samuel Franks, Amy
   University of Pittsburgh trumpet section as they her-         Colcher, Betsy Gettig, Alex Andres, Peggy Polito.
   ald the opening of the HDSA convention!                               HDSA would also like to thank the University
   M e e t t h e HD S A B o a rd o f Tru s t e e s - on Friday   of Pittsburgh for their help in identifying speakers,
   evening, from 5:30 to 6:30 p.m. convention atten-             assisting in the HDSA Activity Center, and participat-
   dees are invited to an informal session with current          ing in our Opening Ceremony.

                                        NYA Co nv enti o n New s
        arlier this month the                fund please go to:                       at www.hdsa.org/nya.
   E    Huntington’s Disease Society
   of America’s National Youth
                                             www.hdsa.org/nya.
                                                     The HDSA NYA is also
                                                                                      Membership is Free.
                                                                                              Includes: One night stay
   Alliance announced the winners            pleased to announce its                  at the Omni William Penn Hotel
   of the 2008 HDSA NYA                      inaugural NYA Day that will              in Pittsburgh, PA, Fun activity
   Convention Scholarship. With a            take place on Thursday, June             on Wednesday Night (June 4th),
   record breaking 25 scholarships           5th at the Onmi William Penn             workshops and a fun-filled day
   distributed this year, the NYA            Hotel in Pittsburgh, PA. There           on Thursday, June 5th.
   looks forward to raising addi-            will be a full day of program-           Deadline to register: May 1,
   tional funds at the National              ming and activities specially            2008
   Convention and throughout the             designed for the National Youth                  For more information:
   year so that they build up the            Alliance. This event is open to all      please go to www.hdsa.org/nya
   fund for the following year. If           registered members of the                or contact Mynelly Perez at
   you are interested in making a            National Youth Alliance (NYA).           (212) 242-1968, ext. 214,
   donation to the scholarship               Not an NYA member? Join today            (mperez@hdsa.org).


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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


   Co ngress P asses Geneti c I nf o rmati o n No ndi sc ri mi nati o n Ac t
        DSA is very happy to              stalled in the Senate after it was     fear led people to be less proactive
   H    announce that on the after-
   noon of April 24 the U.S. Senate
                                          passed in the House in April of
                                          2007 by a vote of 420-3.
                                                                                 about managing their health and to
                                                                                 opt out of participation in clinical
   voted 95-0 in favor of the Genetic     Fortunately for all of us, that was    trials and research studies that had
   Information Nondiscrimination Act,     not the case.                          the potential to bring new thera-
   or GINA (S. 358). The legislation               Over the past year, genetic   pies for their disease. There were
   aims to protect people with or at      disease organizations, medical pro-    many instances where people who
   risk for a genetic disease from        fessionals and Americans across        did reveal their gene status or risk
   discrimination in the workplace or     the country have pressured the         for an inherited disease were
   by a health insurance provider.        Senate to bring GINA to a vote.        bypassed for promotions, fired
   This bill clearly protects people      The result was an impressive           from work, subjected to higher
   who participate in clinical trials     amount of support for the bill and     health insurance premiums or to
   from discrimination as well.           momentum toward seeing it              full termination of their benefits.
            We welcome this victory, as   passed. At the time of the vote        Some state laws provide limited
   it has been more than a dozen          GINA had 46 sponsors. Everyone         protection from such abuse, but
   years in the making. Thirteen          who talked to their Congress           the passage of GINA provides the
   years ago the first version of GINA    members about the need for this        first comprehensive, federal
   was introduced into the House by       bill and encouraged their support      protection from such discrimina-
   Louis Slaughter, a democratic          had a hand in this victory.            tion from which all Americans will
   Representative from NY. It has                  The passage of GINA will      benefit.
   reappeared in every Congress           greatly benefit people living with              GINA still must be signed
   since. Despite broad support for       or at risk for a genetic disease.      into law by President Bush, who
   the bill, GINA was never passed in     For years, in the absence of           has stated his support for the bill.
   both the House and the Senate          federal protections against genetic             With overwhelmingly sup-
   during the same session. Many          discrimination, people have lived in   portive votes in both chambers of

                        May is HD Awareness Month
   feared the bill was destined for
   that familiar fate in this Congress
                                          fear of suffering discrimination
                                          both in the workplace and from
                                                                                 Congress, we anticipate GINA to
                                                                                 sail through the signing ceremony,
   as well when action on GINA was        their insurance providers. This        which is expected soon.

                                            Support the HDSA National Youth Alliance Convention
                                            Scholarship during the month of May.

                                            From May 5th-May 19th place your bids on this hand
                                            numbered and signed lithograph by
                                            Anthony Bennedeto/Tony Bennett.
                                            Lithograph measures 20x16 inches and is framed.

                                            To bid please go to www.hdsa.org/NYA

                                            All proceeds from this auction will go to the HDSA NYA
                                            Scholarship Fund.



HELP FOR TODAY. HOPE FOR TOMORROW.
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


                     Great Events, Great People and
                       Great Hope for the Future
  (continued from page 1)
  and recognizing that we all must work together to
  achieve the success we strive for.
           I am looking forward to the event that brings
  the greatest number of us together, our 23rd Annual
  Convention, in Pittsburgh, this June. It’s a time to
  catch up and look forward; to learn about the latest
  advances from our investigators, our clinicians and our
  local leaders. On Thursday, the day before the
  Convention opens, we have three special pre-conven-
  tion sessions: One for Chapter Leaders, where we
  unveil plans for the upcoming year and discuss issues
  of importance to all; A professional training session
  for our Social Workers, from the Chapters and the
                                                             The St. Louis Chapter’s Left, Right, Center Dice
                                                             The St. Louis Chapter’s Left, Right, Center Dice
  Centers of Excellence, who get to share best practices
                                                             Tournament was a great success. It’s a “pre-
                                                             Tournament was a great success. It’s a “pre-
  and plans for the coming year; and this year, for the
                                                             event” fundraiser to support their wonderful John
                                                             event” fundraiser to support their wonderful John
  first time, a NYA (National Youth Alliance) Day, where
                                                             Turner Memorial Walk for Huntington's Disease.
                                                             Turner Memorial Walk for Huntington's Disease.
  50 of our future leaders will convene to discuss how
  they may increase their role in the Society, and how
  the activities of their organization can be expanded. I
  would like to specially thank Don Barr for his support    scholarships to members who would otherwise not be
  and participation in the creation of this event.          able to attend this important event. I know that many
           I am also very proud to announce that in mid-    chapters are also sponsoring scholarships for NYA
  April the NYA was able to bestow 25 Convention            members and chapter members, and I applaud your
                                                            efforts, and your support of your fellow community
   Belle Plaine, MN Dinner attendees decide which
                                                            members.
   of the many auction items they are going to bid
                                                                     We’ll be talking about several new programs
   on.
                                                            at the Convention, including plans (already underway)
                                                            to expand our Walk-a-thons and other grass roots
                                                            initiatives that will give our chapters and affiliates
                                                            more tools to help with their fundraising efforts.
                                                                     We are also developing several new research
                                                            and care initiatives, as part of the HDSA Board of
                                                            Trustees strategic planning process. These include
                                                            programs to promote upcoming clinical trials, and the
                                                            establishment of Standards of Care for people with
                                                            Huntington’s Disease as a reference for our families
                                                            and medical professionals as well.
                                                                     I hope to see each and every one of you in
                                                            Pittsburgh.




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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



    H D S A Ari zo na R etreat f o r P eo pl e w i th H D Enters Ni nth Year
            Co ngratul ati o ns to Geo rge and M o ni ta Ti mmo ns
                        f o r thei r V i si o n and S uppo rt
        DSA is pleased to announce the 9th annual         the retreat for the past several years.
   H    retreat for Huntington’s Disease. The two day             If you need financial assistance or have any
   event will take place May 30 – June 1 in Prescott questions, please contact Kendall Aitchison at the
   AZ at the lovely Chapel Rock Conference Center         HDSA Rocky Mountain Regional Office, 877-740-
   which is handicapped accessible.                       4372 or kaitchison@hdsa.org. Additional informa-
           Last year more than 134 family members         tion and a registration form can be found on the
   including five affected by juvenile onset HD           HDSA national web site at www.hdsa.org.
   attended the event. This                                                              Don’t miss this
   year’s retreat will feature           “This whole weekend was a life opportunity to participate
   guest speakers Lawrence               changing experience for me.”                and meet other families
   Stern, M.D. from the                       -- 2007 Retreat Attendee               dealing with Huntington’s
   University Medical Center                                                         Disease and juvenile HD in
   in Tucson AZ, Holly Shill,                                                        the peaceful and casual
   M.D. from the Cleo Roberts Center for Clinical         setting located in the cool pines just 1-½ hours
   Research, Helen Brewer, care advisor for Juvenile      north of Phoenix.
   HD in the UK, and Jody Goldstein from the HDSA                 For news of other HDSA retreats visit the
   Center of Excellence at the University of California   national website.

                                       Alyson Krivanek (top left) and daughter Jacy (bottom left),
   San Diego.

                                       Jordan West (top middle) and daughter Stacy, (bottom right),
           The retreat begins at

                                       and Morgan Rott (Volunteer and daughter of Jeanne Rott with
   noon on Friday, May 30,

                                       Mesa vista Care Center in Boulder), attended the retreat last
   2008. There will be a certified

                                       year.
   nurse in residence in the event
   of emergency. The retreat
   costs $150 per adult based on
   double occupancy and includes
   accommodations for two
   nights and 7 meals. Children
   under the age of 10 are free.
   Thanks to the continued
   generosity of George and
   Monita Timmons, an Arizona
   couple, HDSA has a limited
   number of scholarships
   available for people with HD
   and their caregivers.
           It should also be noted
   that a special thank you is
   owed to Phil Hardt, who has
   stepped down after managing
   the logistics and program for




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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



                    C H A P T E R                                          N E W S

  The A ri zo n a Affiliate of HDSA is having its 5th Annual   form with a check made out to HDSA to:
  "Power to Cure" HD Lake Roosevelt Motorcycle Poker                  New England Regional Office– HDSA
  Run on May 10th! If you or any friends                              6 Courthouse Lane, Unit 12
  want to participate in our exciting ride please go to               Chelmsford, MA 01824
  http://www.firstgiving.com/power2curehd or contact           (The website also instructs you on how to register
  Kendell Aitchison (kaitchison@hdsaorg). All of the           online with a credit card)
  money raised will go to defray expenses for our 9th
  Annual Arizona Affiliate of the HDSA, Huntington's           June 29, 2008 is the date for the Bikers for HD
  Retreat this year which will be May 29-June 1st. last        2008 Poker Run. Registration will be from 9AM to
  year.                                                        11AM, and there will be a Pig Roast and Party at
                                                               Knuckleheads Café in Moodus CT at 3PM. Join us
  On Saturday, May 31, 2008, from 5:30 to 9:30, the            again for an event you won’t want to miss. Last year
  Huntington’s Disease -- A Family Story -- Benefit            we had Kevin Hogan, news anchor of Channel 3, join
  Dinner will be held as a tribute to Fred Maher by the        us in the ride. He will be back again this year. $20 for
  Maher Family at Bantam Fire Station in B a n t a m CT. It    Bike & Rider, $10 per passenger, $10 for Pig roast
  will feature a delicious roast beef dinner as well as        and party. Rain or Shine. Contact Sue McGann or go to
  many Chinese auction items. Tickets are $30.00. Go to        www.hdsa-ct.org for more info and to volunteer.
  www.curehd2006.com for more information.                             The Co n n e c t i c u t affiliate is also planning a
  Sponsorships available.                                      eam Hope Walk for a Cure on May 17th at Harkness
                                                               Memorial State Park. Contact Sue McGann to volun-
  The W i s c o n s i n State Conference will be held in       teer or for more info.
  Milwaukee on Saturday, August 23rd. The chapter
  would like to see as many people attend as possible          The Washington University School of Medicine and the
  this year, especially youth! There will be a very nomi-      HDSA S t . L o u i s Chapter are co-sponsoring this year’s
  nal fee (probably only about $5) per person, but it will     regional HD Conference, to be held Saturday, June 21,
  be a great opportunity to learn more about the latest        2008 in Clayton, Missouri from 9-3. It is free of
  news in HD Research and Care, and an opportunity to          charge, including lunch, and will feature the following
  make new contacts. Could you guys spread the word            topics:
  and have youth (and adults) save the date?                           Living Positively with Chronic Illness
                                                                       HD 101
  The N e w E n g l a n d Region is happy to announce the              Driving Assessment
  return of the (2nd Annual )Boston Pub Crawl                          Financial Planning for Long Term Needs
  Crawl For a Cure, Saturday, June 14, 2008. The                       Caregiver Discussion
  Crawl Begins at 1:00 PM. Early Bird Registration (on-                Pre-Symptomatic Testing: Issues, Timing,
  line or postmarked by May 19, 2008) is only $15.00.                            and Whether or Not to Test
  Regular Registration (on-line or postmarked by June                  Social Security Disability Benefits
  1, 2008) is $20.00. To register at the door will cost                Family Planning Options
  $25.00. You must be 21 or Older to register. Go                      Maintaining Independence at Home
  www.hdsa-ne.org and on the Home page find 2nd                        Research in HD
  Annual Boston Pub Crawl. Click on read more.                         Ask the Professional (Panel Discussion)
  Download Registration Form with Waiver.                      Our keynote speaker will be Missouri State
  Both the Registration Form and the Waiver MUST be            Representative Dr. Charles Portwood.To register,
  filled out and signed to participate. Send the completed     contact the HDSA St. Louis Chapter at 314-647-HDSA.




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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




           U P C O M I N G                                                   E V E N T S
   Th u rs d a y, M a y 1, 2008                                   S a t u rd a y, M a y 31, 2008
       Thaw Fashion Show- Northwest Chapter 6:30 PM                    Team Hope Walk - Wisconsin Chapter 9:00 AM
   S a t u rd a y, M a y 3, 2008
        HD Seminar- Upstate New York 8:00 AM                      S u n d a y, J u n e 1, 2008
        Hoop-a-thon - Iowa Chapter 8:00 AM                             Meet the Sopranos - Greater New York Region
        Hoop-a-thon - NJ Chapter 9:00 AM                               5:00 PM

   S u n d a y, M a y 4, 2008                                      r
                                                                  Fri d a y, J u n e 6, 2008 - S u n d a y, J u n e 8, 2008
        Hunt for the Cure- Upper-Midwest                              23rd Annual Convention
        Region 8:00 AM                                            M o n d a y, J u n e 9, 2008
   S a t u rd a y, M a y 10, 2008                                    Swinging for a Cure III - MA Chapter 7:30 AM
        Motorcycle Poker Run- Arizona Affiliate 8:00 AM
                                                                  S a t u rd a y, J u n e 14, 2008
   S a t u rd a y, M a y 17, 2008                                      Walk-a-thon - Rhode Island Affiliate 10:00 AM
        Team Hope Walk- Connecticut Affiliate 9:00 AM                  Pub Crawl- MA Chapter 1:00 PM
        Hoop-a-thon - Indiana Chapter 9:30 AM
                                                                  S a t u rd a y, J u n e 21, 2008
   S u n d a y, M a y 18, 2008                                         St. Louis Family Conference - 9:00 AM
        Team Hope Walk- Illinois Chapter 9:00 AM
                                                                  S a t u rd a y, J u n e 28, 2008
   Tu e s d a y, M a y 20, 2008                                        Walk-a-thon - Vermont Affiliate 10:00 AM
       Gala Gone Wild- Rocky Mountain 6:00 PM                          Team Hope Walk- Wisconsin Chapter 9:00 AM
   Th u rs d a y, M a y 29, 2008 - S u n d a y, J u n e 1, 2008
       Juvenile HD Retreat- Arizona Affiliate

   On April 23rd HDSA volunteers visited the Iowa State Senate. . In attendance (from l to r) were Maiga Krichau,
   Lisa Kirchau, HDSA Regional Development Director Leigh Peterson, Senator Thomas G. Courtney, Greta Peterson,
   Cindy Walker and Brian Walker. Senate Resolution 163 officially recognized May as HD Awareness Month and
   passed unanimously.




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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




                                  Huntington's Disease Society of America


          Win a
          BMW

                Help Us
         Drive Toward A Cure.
        Grand Prize:                                                                    Sweepstakes Limited to only 2,500 tickets sold!
        2008 BMW 328i Coupe                                                             Remember for every two tickets that you buy
                                                                                        or sell for HDSA, you receive a third ticket FREE.
        or $25,000 CASH                                                                 Tickets are $100 each (three for $200).
        Second Prize:                                                                   Remember that all proceeds will benefit
        $5,000 CASH                                                                     HDSA Research Programs.
                                                                                        RSVP Immediately!
        Third Prize:                                                                    Winners will be drawn at HDSA’s National
        $2,500 CASH                                                                     Convention on June 7th in Pittsburgh, PA.

        Fourth Prize:                                                                   Good Luck!
        $750 CASH                                                                       Log on to www.hdsa.org to learn more about
                                                                                        this year’s BMW Sweepstakes.
        Fifth Prize:
        $500 CASH                                                                       For tickets and more information call
                                                                                        1-800-345-HDSA or e-mail hdsainfo@hdsa.org
        We would like to thank BMW of North America
        for helping to make this annual sweepstakes possible.

                                                                                Official Rules:
        No purchase obligation or test drive necessary. No portion of sweepstakes donation is tax-deductible. No responsibility is assumed for lost, late or
        non-delivered mail. Winners will be selected in a random drawing to be conducted on June 7, 2008. All prizes must be redeemed by September 30, 2008.
        Sweepstakes open only to licensed drivers who are 21 years of age or older and are residents of the United States (except Puerto Rico). Employees of the
        Huntington’s Disease Society of America and employees of BMW of North America, Inc., their retailers, advertising, print and promotion agencies and
        members of their immediate families are not eligible. Winners will be notified by phone and/or mail. Odds of winning are determined by the number of
        eligible entries received. Taxes are the sole responsibility of winners. Sweepstakes is subject to all federal, state and local laws and regulations and is void
        wherever prohibited by law. Entry and acceptance of prize offered constitutes permission to use winner’s name, photograph or other likeness for the
        purpose of promotion on behalf of the Huntington’s Disease Society of America, Inc., unless prohibited by law.




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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



                          I N              T H E               N E W S

                    Amy Harmon Awarded Pulitzer Prize
                      for Series on Genetic Testing
   New York Times Reporter, Amy Harmon, was cited by
   the Pulitzer Prize Committee for her ongoing series
   on DNA and Genetic Testing. The Pulitzer Committee
   citation "for a distinguished example of explanatory
   reporting that illustrates a significant and complex
   subject, demonstrating mastery of the subject, lucid
   writing and clear presentation, ..." went on to explain
   that the award was given "for her striking examina-
   tion of the dilemmas and ethical issues that accompany
   DNA testing, using human stories to sharpen her
   reports."
           Amy spent over a year covering Katie's decision
   to undergo genetic testing, her reaction to the results,
   and Katie's plans for the future. The article generated
   great awareness of the issues of testing for the mutat-
   ed huntingtin gene, and how family members face the
   specter of Huntington's Disease. The story led to addi-
   tional national coverage on "The View," and in other
   print and video media.
           Barbara Boyle congratulated and thanked Amy
   on behalf of the entire HD community. If you would
   like to add your wishes and congratulations, please
   send an email to amy@hdsa.org. We will forward all
   these messages to Amy Harmon.

   NY Times article on Katie Moser by Amy Harmon (r)




       The
       Pacin’
       Parson
       Walks for
       HDSA
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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


                                  R E S E A R C H
   D rugs w hi c h i nduc e auto phagy are added to the researc h pi pel i ne
     nducing autophagy as an alternate way to enhance        the UPS in case the theory proves to be true.
   I the clearance of the HD protein is a very promising
   strategy for preventing or delaying the onset of
                                                             Inducing autophagy as a treatment strategy
                                                             Whether or not the UPS itself is impaired in
   Huntington's Disease.                                     Huntington's Disease, there is good evidence to
           A new study by Professor David C. Rubinsztein suggest that it is not capable of handling the HD
   and colleagues suggests new drugs that induce             protein and that its clearance is mediated by an
   autophagy for the research pipeline. Protein clearance, alternate method called autophagy.
   the UPS, and HD.                                                  Autophagy literally translates as 'self eating.'
           After a protein is made, it needs to be folded    In this very old cellular house cleaning process (it's
   correctly to be able to do its work in the cell.          found in organisms from yeast to mammals),
           This doesn't always happen and misfolded          damaged parts of the cell, pathogens, and large
   proteins are targeted for degradation. The normal         proteins are surrounded and consumed.
   huntingtin's protein is degraded                                                   Autophagy is even induced
   though the ubiquitin proteosome                                                  during times of starvation—less
   system (UPS). In the ubiquitin                                                   essential parts of the cell will be
   proteosome system, proteins                                                      consumed for nutrition. This may
   which are not needed or                                                          explain why caloric restriction
   which have misfolded are tagged                                                  initially helps the HD mice,
   for degradation by a small protein                                               because it induces autophagy.
   called ubiquitin. The unwanted                                                     A potential treatment strategy
   protein is then moved into the                                                   would be to safely induce
   proteosome, a barrel like protein                                                autophagy to enhance the
   complex, which breaks it down                                                    clearance of the HD protein.
   into amino acids that can then be                                                The one known way to induce
   recycled.                                                                        autophagy with a medication has
           One theory holds that the                                                been through rapamycin, an old
   UPS is impaired in Huntington's           Professor David C. Rubinsztein
                                             Professor David C. Rubinsztein
                                                                                    antibiotic. Professor Rubinsztein
   Disease. If that is true, it could                                               and his team have done extensive
   explain quite a bit about HD                                                     research on rapamycin and
   pathology because the UPS appears to play an impor- autophagy.
   tant role in many regulatory processes in the cell.                       However, researchers have been
           This theory is controversial, however. Some       reluctant to bring rapamycin to clinical trials because
   labs using certain methods have found evidence to         the drug would need to be taken over many years
   suggest that it is and others using different methods     and it has side effects at the levels needed for it to
   have not. It is also possible that the conflicting find-  be effective, one of which is suppressing the immune
   ings can be explained because the researchers were        system. The search has been on for alternatives and
   looking at different points in the disease process (see   in an exciting new study, Professor David Rubinsztein
   http://www.pubmedcentral.nih.gov/articlerender.fcgi?t and colleagues report that several drugs that are
   ool=pubmed&pubmedid=18047739 for a good                   already FDA approved for other purposes also
   review of the issues).                                    stimulate autophagy.
           The UPS is an important line of investigation             "By screening a number of drugs that have
   because of the potential for treatment and                already been shown to be safe in humans, we have
   researchers are already looking into ways to target       (continued on page 11)



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   D rugs w hi c h i nduc e auto phagy are added to the researc h pi pel i ne

   (continued from page 10)                              method of protein clearance isn’t working in HD.
   been able to identify some unexpected and very        Inhibiting calpain induces autophagy.
   promising pathways involved in Huntington's," says            One treatment possibility the research team
   Professor Rubinsztein, a Wellcome Trust Senior        suggests researching further is a combination of one
   Clinical Fellow at the University of Cambridge. "In   of the drugs identified in the study and rapamycin
   collaboration with Cahir O’Kane’s group in Cambridge since they work by different pathways. The idea is
   and Summit Plc, we have shown that these drugs can that a lower dose would be needed for each,
   alleviate the toxicity of the Huntington’s disease    furthering reducing potential side effects.
   mutation in cell-based, fly and zebrafish models. The         The next step is to test the drugs in mouse
   big question for us is whether they will do the same  models. If one or more of the drugs identified are
   in humans."                                           shown to be effective in an HD mouse model, the
            The research team screened 256 existing      following step would be clinical trials with people.
   drugs in use for other                                                                Since both verapamil and
   medical conditions and        "If we can find a safe, well tolerated drug, clonidine are FDA
   found several which           then a person at risk could be placed on a              approved for other pur-
   induce autophagy. Two         drug regime to help prevent onset. It is                poses and have a known
   drugs are of particular                                                               safety profile, the clinical
   interest, verapamil and
                                 much easier to stop something happening trial process would be
   clonidine. Verapamil is       than having to treat it once it has started." shorter than the process
   prescribed for high blood                    — Professor Rubinsztein.                 of trying a newly devel-
   pressure and many                                                                     oped drug. Although
   people take it for years.                                                             much work remains to be
   It is an L-type calcium                               done, the results so far
   channel antagonist and stimulates autophagy by        are exciting. "We know the genetics of Huntington's
   reducing the influx of calcium into cells. Calcium    disease and can predict the majority of people at
   handling is known to be a problem in HD.              risk," says Professor Rubinsztein. "If we can find a
            Clonidine is prescribed for migraines. It is safe, well tolerated drug, then a person at risk could
   a regular of inositol trisphosphate (IP3) levels. It  be placed on a drug regime to help prevent onset. It
   induces autophagy through the reduction of cAMP.      is much easier to stop something happening than
   cAMP, which stands for cyclic adenosine monophos-     having to treat it once it has started."
   phate, is a molecule which is responsible for a                         - Marsha L. Miller, Ph.D.
   number of functions in the cell, including regulating
   the passage of calcium through ion channels. cAMP     References:
   is known to be elevated in HD.                        Janet E Davies, Sovan Sarkar, and David C Rubinsztein.
            Although these drugs work differently, they  "The ubiquitin proteasome system in Huntington's disease
   both affect different parts of the same cyclical      and the spinocerebellar ataxias." BMC Biochemistry 2007
                                                         Nov 22;8 Suppl 1:S2.
   pathway in which cAMP regulates IP3 levels which
                                                         Andrea Williams, Sovan Sarkar, Paul Cuddon, Evangelia K
   increase calpain activity, which cleaves and then
                                                         Ttofi, Shinji Saiki, Farah H Siddiqi, Luca Jahreiss, Angeleen
   activates Gs alpha, which in turn regulates cAMP      Fleming, Dean Pask, Paul Goldsmith, Cahir J O'Kane,
   levels. Intervention at any point in the process was  Rodrigo Andres Floto & David C Rubinsztein. “Novel tar-
   shown to be effective in inducing autophagy in the    gets for Huntington's disease in an mTOR-independent
   study. Caplain is elevated in HD and appears to       autophagy pathway.” Nature Chemical Biology published
   inhibit autophagy which may be why this alternate     online 23 March 2008.




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                  2008 HDSA National C onvention Program
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                  Meet Your HDSA Regional Staff
                  H D SA N e w E n g l a n d R e g i o n




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