From Natalie by wuyunyi

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									Natalie’s Wish
                                                                                                     Star light, star              bright,
                                                                                                     First star I see             tonight
                                                                                                     I w i s h I m a y, I         wish I might
                                                                                                     Have the wish,               I wish tonight.


                                                                                                    Dear Friends and Family
                                                                                                    In February 2003, on Natalie’s twelfth birthday,
                                                                                                    she shared her secret wish with me: “To have
                                                                                                    my disease go away forever.” Because of all
                                                                                                    of you, that wish is one step closer to becoming
                                                                                                    a reality. Our fourth annual Natalie’s Wish event
                                                                                                    on June 2, 2005 was an extraordinary success.
                                                                                                    This amazing evening began with best-selling author
                                                                                                    and journalist, Morton Kondracke sharing the story
                                                                                                    of his late wife Milly’s courageous battle with
From Natalie                                                                                        Parkinson’s disease. Mr. Kondracke emphasized
                                                                                                    the need for funding medical research especially
                                                                                                    for orphan diseases like Cystinosis.
   y      I started 9th grade in September. I recently started
   y                                                                                                After Mr. Kondracke’s presentation, we watched
   y      playing basketball for my high school JV team. So far,                                    in amazement as our first live auction raised
   y      it is my favorite part of high school!                                                    $98,000 for Cystinosis research. Following
                                                                                                    the auction, the highlight of the evening was
Last year, I started a new medication for my eyes. In October I had an appointment
                                                                                                    the Fund-A-Cure segment where we again were
at the National Institutes of Health (NIH) in Bethesda, Maryland. I met with Dr. Tsilou             astonished as more than $290,000 was donated for
because my eyes always felt itchy and sometimes it felt like I had an eyelash in my                 research. By the end of the night, over $825,000
eyes even though I didn’t. Dr. Tsilou is the doctor who examines the eyes of kids                   was raised for Cystinosis research. 100 percent
with Cystinosis. I had an early morning appointment at the NIH. They examined                       of every dollar donated to the Cystinosis
my eyes, dilated them and took pictures of the crystals covering my corneas.                        Research Foundation goes to Cystinosis
                                                                                                    research. All costs of the event and day-to-day
The doctor saw lots and lots of cystine crystals in my eyes. That was not a good
                                                                                                    costs of the foundation are completely underwritten.
thing because if more crystals form, I could lose my eye sight or damage my                         In 2005 alone more than $1,146,000 was raised
corneas. That would be awful and it would make it hard to live a normal life.                       for research!
When I found out that I could go blind if I did not take the eye drops, it scared
me and I knew I had to use the eye drops every day.                                                    We could not do this without all of you.
The doctors told me that I had to take the eye drops 8 –12 times every day or else                      Your outpouring of love and support
the cystine crystals would keep forming in my eyes. If I don’t take them regularly
                                                                                                               has been invaluable.
the crystals won’t go away. If I take the drops all the time, the crystals will eventually
dissolve but the hassle is that I have to take them every day forever. I am glad there              We cannot thank you enough for your support in our
are eye drops and I have a treatment. I have learned to take the eyedrops by myself.                efforts to find better treatments and ultimately a cure
Thank you everyone for making such                                                                  for Cystinosis. Your support has allowed us to fund
                                                                                                     a number of multi-year research studies, something
a difference and helping all of us with
                                                                                                    that had been impossible in the past due to lack of
Cystinosis have hope for a better future.
                                                                                                    long-term funding. Currently there are six research
                                                                                                    studies underway and one study being reviewed and
                                                                                                    pending approval by the Scientific Review Board.
                                                                                                    We look forward to making an announcement soon!

                                           Alex and Natalie Stack                                   continued on page two
BOMA Golf Tournament for                                                                                       I am inspired by
Cystinosis Research Foundation                                                                                  my good pal Natalie...
For the second year in a row, the Building Owners and Managers
Association (BOMA) of Orange County chose the Cystinosis Research
Foundation as the beneficiary of their Annual Charity Golf Tournament
in August, 2005. The tournament sold out with 144 participants.
The live and silent auction raised over $9,000 for the Foundation.
                                                                                                                 David Martin
We are grateful to our friends at BOMA-OC. Special thanks to the                                                 Tri-ing to
committee who worked so tirelessly: Bob Brans of Structural Materials,
John Czorniak of Pacific Building Care, Diana Dean of Environomics                                               Find a Cure
West, Tom Devlin of Allied-Barton Security Service, Dale Fedele of
Ampower, Seth Foster of HSG/Birdbusters, Lynn Infurchia of Belfor                                                 for Natalie
USA, Jeff Koscher of Advanced Restoration, Lisa Miller of CarrAmerica
Realty Corp., Monique Mora of the Gale Company, Betty Pickett of                                All of us have ideas about certain limitations
Universal Protection, Mike Raring, AAA Electrical & Communications,      and restrictions and what we can and cannot do. Often we hear “shouldn’t”
Jon Schneider of Specialty Uniform Services, Eric Sorensen of Able       and “couldn’t” and use those terms for not trying at all. I am inspired by
                    Engineering, Brett Wells of Frazee Paint and         my good pal Natalie who rejects those negatives and says “Yes I can.”
                      Robin Jochims of BOMA-OC. Thank you!               So in spite of the “shouldn’t” and “can’t”...I “could” and “did” complete
                                                                         the Lavaman Triathlon in Hawaii. Again spectators asked “who is Natalie?,”
                        The 2006 BOMA-OC Charity Golf Classic
                                                                         “What is Cystinosis?” I was so proud to share Natalie’s wish.
                           will be held August 2, at the Talega
                             Golf Club in San Clemente. For details      Thank you Natalie for allowing me to share your story
                             visit          and represent the Cystinosis Research Foundation.

Dear Friends and Family continued from front page
The Cystinosis Research Foundation (CRF) is the largest nonprofit        refrigerated and must be taken every
funder of Cystinosis research. As of 2003, the CRF has funded more       waking hour a minimum of eight times
than $1.5 million supporting seven Cystinosis Research projects.         a day. The drops dissolve the crystals but
This year more than $895,000 is available for Cystinosis research        they must be taken for the rest of her life or the crystals will reaccumulate.
projects. We will soon announce to the medical and scientific            Although the eye drops sting and smell, Natalie has learned to take them
communities that we are accepting grant proposals. On June 1,            herself. We are grateful there is a treatment and that she is able to tolerate it.
during our Natalie’s Wish event, we will announce which research
                                                                         It is not just our family that is so thankful. We receive letters from other
studies will be funded in 2006.
                                                                         families who have children affected by Cystinosis. So many of their children
We are continually amazed at how blessed and fortunate we are to         are very ill and suffering every day. They thank us for the new and continuing
have such a wonderful community of family and friends. We could          research funded by the CRF. It is because of all of you – our community of
not do this without all of you. Your outpouring of love and support      friends and family that have made a difference. So, on behalf of all of the
has been invaluable and we treasure every letter, email and note         families with Cystinosis, we are eternally grateful for your continued support
we receive. Your prayers have made a difference – we are moving          as we move closer to improving and prolonging the lives of our children.
into the next clinical phase of the slow-release medication study.
                                                                         Your financial support has provided Natalie and others with Cystinosis the
Research is slow but we are continually learning new and important
                                                                         hope they so desperately need. We thank you for your commitment to our
information about Cystinosis. It is a time of hope!
                                                                         cause. We are also grateful to the many volunteers who made our Natalie’s
Although Natalie looks healthy on the outside, inside she is not.        Wish event such a success. We are especially thankful for the tireless efforts
In October we visited the NIH because she was experiencing pain          of Zoe Solsby and Marylyn Milburn.
and discomfort in her eyes. The exam revealed that Natalie’s corneas
                                                                         We hope that you will join us for this year’s Natalie’s Wish event on Thursday,
were covered with cystine crystals. Accumulation of the crystals
                                                                         June 1, 2006 at the Balboa Bay Club in Newport Beach. The evening promises
causes photophobia and pain similar to the feeling of having sand in
                                                                         to be inspirational and uplifting with Kevin Sharp, an award-winning vocalist
your eyes. Although the news was not good, it was not unexpected
                                                                         and speaker, sharing The Power of A Wish – his story of hope and survival.
given the inevitable progression of Cystinosis. Fortunately, there are
                                                                         We look forward to seeing you there.
experimental eye drops available as a treatment. We are now in our
fourth month of eye drops. To be effective, the drops need to be         Nancy and Jeff Stack
                                        Feature on Sigma-Tau
                                            “What’s the difference between a disease affecting 300 and 300,000,000? To us, nothing.
                                            At Sigma-Tau Pharmaceuticals, Inc., our mission is to help ensure patients with rare diseases are not left
                                            untreated. We are dedicated to providing new therapies and new hope for patients with rare diseases.”

Laura’s a Picky Eater                                                                       Laura’s success with her treatments
                                                                                            was due to the dedication of her
Marybeth knew that there was something wrong with her daughter Laura                        parents, clinical researchers and
almost from birth. She was a picky eater with cravings for salty and spicy                  companies like Sigma-Tau, who are
food, and she was so tiny for her age that her measurements didn’t make it                  dedicated to research for treatments for rare diseases like Cystinosis.
on to the growth charts. But it wasn’t until her second birthday – after being              Laura’s participation in the clinical study, helps other children with
admitted to the hospital for severe dehydration following the flu – that doctors            Cystinosis be assured that there is hope.
finally consulted Dr. Frederick Kaskel, a pediatric kidney specialist.
As soon as he heard Laura’s story, Dr. Kaskel suspected Cystinosis. Laura’s
parents weren’t aware at the time, but he had recently attended a lecture
                                                                                            Life Is Looking Up for Jack
by Dr. William Gahl of the National Institutes of Health, who had described                 Can you imagine watching a three-year-old child guzzle an entire 20-ounce
Laura’s problem. After a battery of tests, Dr. Kaskel confirmed Laura’s                     bottle of water in one gulp? Or watching your son vomit up everything he
diagnosis of Cystinosis, and her parents were on the phone talking to                       eats? His parents, Christy and Dave can.
Dr. Gahl, who had dedicated his life’s work to this rare disease.
                                                                                            When he was born, Jack was a big, happy baby who ate, cried, slept and
After the conversation, Laura’s parents knew they were in capable hands.                    played much like his older sister. As he grew, however, instead of walking
Marybeth recalls, “Instead of saying, ‘Don’t worry about her, she’ll probably               like other kids, Jack continued to scoot across the floor on his bottom. His
outgrow this’, he asked questions and listened to my answers.” She learned that             parents also noticed that he drank a lot and went through a lot of diapers.
Cystinosis is an inherited metabolic disease that results in an abnormal                    But he was a big baby, so his parents just assumed it was because of his size.
accumulation of the amino acid cystine in organs of the body. This can
                                                                                            When Jack stopped eating and started vomiting regularly his parents knew that
cause many serious complications. An oral treatment called Cystagon® can
                                                                                            something was wrong. Visits to their pediatrician led to consultations with
help manage the crystal accumulation in all the organs except the eye.
                                                                                            gastroenterologists, geneticists, nephrologists and ophthalmologists. As a
Tiny crystals of cystine begin to develop                                                   result, his parents, Christy and Dave, got a crash course in obscure diseases.
in the cornea and cause hypersensitivity
                                                                                            Since that diagnosis at one-year-old, perspective became the battle cry of
to light that Laura experienced. The tiny
                                                                                            their daily lives with Cystinosis, and the word ‘normal’ has taken on new
specks shown in the bar of light are
                                                                                            meaning. Jack now has a gastric feeding tube in his stomach through which
corneal cystine crystals – the result
                                                                                            he receives liquids, nutrition and medicine. He’s also taking a number of
of cystine build-up.
                                                                                            medicines to manage his disease. Jack’s everyday, six-hour medical routine
Not long after meeting Laura and hearing her story, Dr. Gahl asked Marybeth                 has enabled him to grow surprisingly well, and allowed him to become
to enroll her in an eye drop protocol study of children with Cystinosis being               one of the healthiest of all kids with Cystinosis.
conducted at the NIH. Since then, and for most of her life, Laura has been
                                                                                            But despite the lifelong medication regimens and challenges of preparing
involved in testing the experimental eye drops.
                                                                                            for an independent adulthood, patients like Jack can be confident that
The eye drops are being developed by NIH with support from Sigma-Tau                        others will continue researching this disease to add to the knowledge and
Pharmaceuticals, Inc. Sigma-Tau is dedicated to researching and developing                  understanding of how to better manage it, while offering hope to others.
treatments to improve the lives of patients and families living with rare
                                                                                            Dedicating resources to the NIH research is just one way that Sigma-Tau
diseases. Simply because it’s the right thing to do.
                                                                                            demonstrates its commitment to rare diseases. Sigma-Tau has supported
Laura does not want to draw attention to her condition, thus the challenge                  patients with these diseases since 1985, when it became one of the first
of getting a busy young adult to administer the eye drops on a regular basis.               companies in the world to obtain an Orphan Drug approval in the United
The road has not been easy. Every four months for much of her life, Laura                   States. To Sigma-Tau, there is no greater accomplishment than helping
returned to the NIH for monitoring and medication adjustments.                              people with rare diseases. Its mission is to help ensure that patients with
                                                                                            rare diseases are not left untreated.
Now 20, Laura is a normal, young adult. Early on, her family developed a
philosophy for living with the disease: “We can’t change what you have,”                    Sigma-Tau Pharmaceuticals, Inc., is one of those rare companies dedicated
her parents told her, “we have to learn to live with it.”                                   to discovering new therapies for patients with rare diseases.
      Birthday Wishes
Birthday Party
      Benefits Cystinosis
      Research Foundation
       Kelsey Valley, Shelby Searles, Jenny Madden and Caroline Kelter, four Harbor Day School 8th graders, who have been Natalie’s
       classmates for nine years, decided to celebrate their fourteenth birthdays with a party to benefit the Cystinosis Research Foundation.
       On Friday, May 27, 2005, Memorial Day weekend, the girls boarded the TIKI boat out of the Balboa Fun Zone with 45 classmates and seven
       brave Moms. They toured the harbor for five hours while dancing to the sounds of a DJ from Los Angeles who played their favorite songs.
       In lieu of gifts, the girls requested that guests write a check to support Natalie’s Wish and by the end of the evening they
       had raised $6,415! The girls were so thrilled they thought about making this an annual event to help Natalie’s wish come true.

                                                                                         Ava’s First Birthday
Kirsten’s Eighth Birthda ing
                                                                                           By Renee Carter – Ava’s Mo
                      someth                                                              On April 2, 2005 our daught
                                Kirsten Hansen wanted to do                                                               er Ava
For her eighth birthday party,                                  theme.                    turned one year old. A milest
                                a theatre party, with a Grease                                                            one –
special. She decided to have                                     in a                     a time to reflect on the wonde
                                 r lead Kirsten and her guests                                                             rful
A professional actress/directo                                  the end                   progress in her young life –
                               them in 50’s era costumes. At                                                           and for
 rehearsal and then dressed                                      al Grease               us, a time to honor our dea
                                  d a short version of the music                                                      r friend
 of the party the girls performe                                cial part.               and a true inspiration, Natalie
                                s. However, this was not the spe
 for their parents and familie                                 guests each               Stack. When our son, Nathan
                                 to the party, Kirsten and her                                                          , had
  Instead of bringing presents                                   .                      his first birthday in August of
                                   tinosis Research Foundation                                                           2003
  made a contribution to the Cys                                                        we requested “no gifts.” We
                                                                   n wanted                                            also asked that if guests felt
                                   r Day School family and Kirste
   Natalie was in Kirsten’s Harbo                    th          r. More
                                                                                        compelled to give something
                                                                                                                      , they make a donation on his
                                 her during their 8 grade yea
   to do something special for                                   time.
                                                                                        behalf to the Cystinosis Resear
                                                                                                                         ch Foundation.
                                  the girls all had a wonderful
   than $1,000 was raised and                                                          We continue to believe the bes
                                                                                                                         t gift we can give our children
                                                                                       is the willingness to share the
                                                                                                                       ir blessings of good health
                                                                                      and good fortune with those
                                                                                                                      who are less fortunate. For this
                                                                                      reason, we chose to jointly cel
                                                                                                                        ebrate the life our youngest
                                                                                      child, Ava, and the life of Nat
                                                                                                                     alie and others with Cystinosis.
                                                                                     We are honored to include Nat
                                                                                                                        alie and the entire Stack fam
                                                                                     among our most celebrated                                          ily
                                                                                                                    of friendships.
                                                                                     Ava’s party was wonderful. The
                                                                                                                        In-n-Out Burger truck served
                                                                                    our guests; a princess castle
                                                                                                                    jumper provided a fun outlet
                                                                                    the kids; and a face painter                                      for
                                                                                                                   and balloon twister topped off
                                                                                    day. A day that we’ll always rem                                  the
6    Questions for
    Lynette and Michael K. Hayde

    ‚   How did you first learn about the Cystinosis Research Foundation?
            Mike and Lynette knew for many years that Natalie Stack had a rare disease. But they never approached Jeff or Nancy about
            it because they did not want to intrude or cause them any more pain than they were already living with. They did, however,
            respond to an invitation to attend a Natalie’s Wish event where they first learned about Natalie’s disease. It was a tough
            evening for them and for everyone who attended. Mike and the gentleman he sat with “were in tears” much of the evening.

    ƒ   You know the Stacks personally and professionally. Is that why you first became donors?
        Are there other reasons you continue to be so committed?
            Mike has known Jeff for more than 20 years. They are friendly yet fierce competitors in the apartment business. But they
            have a great deal of respect for each other. Their business relationship is almost certainly what got Mike and Lynette to
            attend that first event. “We became donors because we were deeply touched as we listened that night,” Lynette said. While
            Mike had an initial gift in mind, Lynette immediately rejected it and said, “We have to do more.” Lynette is a compassionate
            person who believes in miracles. She also believes, “that if the mind can conceive it, then it can become a reality. As long
            as we are able, we will work to make Natalie’s wish come true.”

    „   With your extensive expertise in the nonprofit world, what distinguishes the Cystinosis Research Foundation
        from other nonprofits? Are there other organizations that have a fundraising approach similar to CRF and
        its commitment to good stewardship?
            Mike and Lynette decided long ago they would spend most of their time and money helping children because “they are the
            future.” “We give to many important causes, but most of the time we don’t personally know those who benefit from our
            giving. The Cystinosis Research Foundation presented us with a unique opportunity. Of course we understand the benefits
            for those who are affected by this terrible disease. But in this case we personally know someone who suffers from Cystinosis.
            Natalie is very special to everyone who knows her. This makes this quest extremely personal.”

    …   Cystinosis is a rare disease affecting only a few hundred children in the United States.
        How big a role does that play in your commitment to the organization?
            “If not all of us, then who will take on this challenge?” asks Lynette. It is clear that the major pharmaceutical companies
            cannot or will not do research on a disease if the market is so small it will not generate a profit. While most of us don’t
            fully understand or even agree with this reasoning it seems that “it’s up to us to make a start. We hope the Cystinosis
            research we’re helping to fund will provide crossover benefits for other diseases. Then the market may be large enough
            to entice a major pharmaceutical company into the fight.”

    †   As advocates for Natalie’s Wish, what would you say to others to encourage them to be involved?
            “We all have our favorite causes,” but Mike and Lynette still ask their friends and colleagues to help Natalie. “This courageous
            young lady knows her life expectancy is short but she is still filled with joy,” Lynette says. Mike and Lynette are convinced
            they can make a difference, and they encourage others to help as well. “If someone can look at Natalie and other children
            with Cystinosis and turn away, there is nothing more we can say to convince them to help,” she adds.

    ‡   You have made a large financial contribution to the CRF for Cystinosis research, what are you thoughts?
            “Some would say that there’s not much chance of the research being successful, but we like siding with the underdog,”
            Mike says. An American president once got school children to collect dimes to find a cure for polio. It was a dreadful
            disease and seemed unconquerable. But in his youth Mike was part of that effort. Today, when the vaccination is given
            there is no polio. Now Mike boldly adds, “We will support Cystinosis research until we find the cure.”

                                                                         Committed to Making a Difference
2005 Donor List                           Donor List
$50,000 AND MORE                          $2,500 AND MORE
Lynette and Mike Hayde                    Randolph and Judith Agley             Jim and Jennifer Damon
                                          Amies Communications                  Roberta and Jerry Dauderman                         Sisters Sarah, age 22,
SARES • REGIS Group                       Dianne and Richard Azar               Yvette del Prado                                         and Katie, age 20
Nancy and Jeff Stack                      Carol Cranfield                       Draper Family Foundation Fund
                                          Louise and Peter Donovan              Anne G. Earhart
                                                                                                                     Dzida, Carey & Steinman
$20,000 AND MORE                          Steve and Susie Duffy,                Dick Eddy
                                                                                                                     Jeff and Stacy Stone
                                             Western National Realty Advisors   Ray Eldridge
Sheri and Chris Dialynas                                                        Bob Elliott                          Janet and Jim Stoneman
                                          Linda and Kris Elftmann
Equity Residential Foundation             J.P. Morgan Chase                     Rick Emsiek                          Barbara and John Stuart
                                                                                Gail and Dale Fasse/RK Advertising   Style Interior Design
David and Suzu Neithercut                 Darby T. Keen
                                                                                                                     Marilyn and Tom Sutton
                                          Mary Ann King                         Favreau Engineering, Inc.
Marcy and Gerry Spector                                                         Patti and Ed Feldman                 Kyle and David Team
                                          Dale Kistler and Sue Murahata
Ginni and Kent Valley                     Karen and Tom Linden                  Janet and Frank Foster               Cassie and Jim Thomas
                                                                                Franciscan Friars                    Leslie and Will Thompson
                                          Eric Luna
$10,000 AND MORE                          Makar Properties, LLC                 Fuscoe Engineering, Inc.             Gary and Marlene Tolfa
Gerson and Barbara Bakar                                                        Edward J. Geraghty                   Nina and John Townsend
                                          Robert and Nancy McLeod
Diane and Bruce Bearer                                                          Tom Gibbs                            Dr. M. Vellard and Dr. E. Kokkis
                                          Susan and Tom Moran
                                                                                Bridget and John Gormly              Nanette and Don Vodra
Renee and Daryl Carter                    Margery and Jeffrey Morgan
                                                                                                                     The Waldeck Family
                                          Doug and Donna Neff                   GoWest Equipment Leasing
Judy and David Christensen                                                      Chuck Groux and Dave Taborelli       Ann and Dave Weinstein
                                          Eric and Shirley Pepys
Fidelity National Title                                                         Beth Anne and Don Haarer             Dori and Richard Werner
                                          RL and David Peters
Jack and Marilyn French                   Bob and Betty Stine                   Kathy and John Hamilton              Ann and Marc Winthrop
Merry and John Hagestad                                                         Dee and Larry Higby                  Arn and Nancy Youngman
                                          Allan and Cindy Stokke
                                                                                Diane and Paul Hurst                 Stephen Zotovich
Allan O. Hunter, Jr.                      Mark Tennison
                                                                                Scott Ingraham                       Ron and Janet Zuzack
John and Jill Manly                       Jill and Larry Tucker
                                          Watson Land Company                   Tracey and Paul Irving
Joe and Lori McKay                                                              Scott and Susan Jackson              $500 AND MORE
                                          Wells Fargo Bank
Kim Megonigal, KIMCO Staffing Services                                          Michael B. Jeffers                   Dana and Jim Abbott
Christyne and Bob Olson                                                         Sherry and Park Kennedy                 and Maddy Abbott
                                          $1,000 AND MORE                                                            Dick and Mary Allen
Oltmans Construction Co.                  Bette and Wylie Aitken                Linda and John Kensey
                                                                                Peter and Valerie Kompaniez          Jon and Bettyjane Anderson
Quentin Primo                             Bill and Annette Albert
                                                                                                                     Antonello Ristorante, Inc.
                                          Ginger and Tony Allen                 Lincoln Consulting Group, LLC
Milan Puskar                                                                    Mary Lonich                          Randall M. Babbush
                                          The Alter Family
Ron and Deborah Ratner                                                          MacFarlane Partners                  Lynn and Art Barrett
                                          David Ash
Red Mortgage Capital Group, Inc.          Bank of America                       Francine and Dominic Magliarditi     Louise and John Basso
RENTNET                                                                         David E. Martin                      Nancy and Dennis Bear
                                          Bemus Landscape, Inc.
                                                                                Duncan and Shirley Matteson          Hal Beral, Golden State Developers
Cyndi and Charles Rinehart                Jill and Blake Bertea
                                                                                Debbie and Kevin McKenna             Marta Borsanyi/
Mary and Peter Rooney                     Lesley and Alan Beyer
                                                                                Carolyn and Gary McKitterick            The Concord Group
                                          Lorraine and Doug Bibby
                                                                                                                     Nancy and Louis Bowen
$5,000 AND MORE                           Lan and Charles Bolus                 Jim and Oralee McNamara
                                                                                Meredith, Weinstein & Numbers        Lyons and Susanna Brown
Ziyad Abduljawad                          David and Judy Breitstein,
                                                                                Nora Miller and Brian Dowd           Kristen Sandifer and
                                             Duke of Bourbon
Allen Matkins Leck Gamble & Mallory LLP                                         William A. Millichap                    Charlie Bullock
                                          Donald Briscoe, Force Framing
Shelley and Phil Belling                  Carolyn and Preston Butcher           Melinda and Tony Moiso               Ken, Julie and Trevor Busch
BOMA Orange County                                                              Constance Moore and Roger Greer      Susan and Wade Cable
                                          Tim Byrne, Lincoln Property Company
                                                                                Ann and Mike Moorhead                Caillouette Family
Ralph Eidem, Jr.                          George Bystrom
                                                                                Don Morrow and Judy Johnson          Diane and Jim Connelly
Don and Sonja Grant                       Craig Cadwalader
                                                                                National Multi Housing Council       Cornerstone Real Estate Advisers, LLC
Mr. and Mrs. James D. Harper, Jr.         Call, Jensen & Ferrell
                                                                                Newkirk Enterprises                  Allen Court
                                          Chapman, Glucksman & Dean
Sharon and Terry Hartshorn                Paula and Jeff Cole                   Augie and Lynne Nieto                Doug and Liz Culkin
Lisa and Brad Hillgren                                                          NWP Services Corporation             John L. Curci
                                          Budge Collins
Susan and Mark Hillgren                                                         Aaron L. Pacillio                    Dan and Judy Daniels
                                          Corpus Christi Catholic
                                                                                Lisa and Barney Page                 Sean P. Deasy
Fritz Hoelscher                              Christian Community
                                                                                                                     Scott Dennis
                                          Con Am Management Corp.               Bob and Bunny Perlberg
LNR Property                                                                    Yvone Perlberg/AMGEN FOUNDATION      Robert DeWitt
                                          Anne Marie and Bob Couglan
John and Janice Markley                                                         James M. Peters                      Stephanie and Bruce Dorfman
                                          Ron Cox
Karin Krogius and Scott Mason             Diane and Gene Crain                  Robert C. Ruocco                     Barbara and Bill Driscoll
Steven Myers                                                                    Denise and Patrick Russell           Jerry and Gail Durkin
                                          Kim and Dick Crawford
                                                                                John Schaefer                        Diane and John Erskine
Chrissy and Rick Nicholas                 Doug and Cindy Crocker
                                                                                Rick Scherrer                        Michael S. Fallman
Sue Ellen and Paul O’Connor                                                     Dennis Schmucker                     Fieldstone Foundation
Steve Quinn and Pam Bellamy                                                     Tom and Jill Schriber                Malin Fletcher
Regis Homes of Northern California                                              Leslie and David Seidner             Bill and Marcia Gaboury
  Rob Wagner & Mark Kroll                                                       Shapell Industries, Inc.             Caroline and James Gapp
Gary Ryness                                                                     Lauren and Bob Silvernail            Stephanie and Jim Gilly
                                                                                Melvin and Bren Simon                Russell and Donna Goodman
Janet Keller and Bernie Schneider                                                                                    John and Judy Goolsby
                                                                                Zoe and Don Solsby
Christine and Bill Thormahlen                                                   Sheila and Ygal Sonenshine           David and Ruth Green
The Ryland Group                                                                Steve Stambaugh & Elena Cappadona    Larry and Debbie Green
Sheila and Ted Weschler                                                         Janet and Mike Starzyk               Tricia and Mark Haarer
                                          Hugh, age 4, diagnosed prenatally
For more information about Cystinosis, visit

Randolph G. Hawthorne               Lara and Peter DiCorpo               Wendy and George Bigelow              Kathleen and Ben Jackson          Diane and Michael Costanzo
Donna and Mike Heiken               Mark Domash                          Dorine and Brandon Birtcher           Mike and Maisie Jara              Keith Covington
Kyle B. Herren, Lockton Companies   Angela and Tim Donahoe               Carole Blake                          Susan and Sean Johnson            Margaret and Raymond Cully
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                                                         Cystinosis Research Foundation
                                                         Research Updates from the Doctors

Corinne Antignac, MD, PhD                        in which the mouse gene has been inactivated.      strains and are currently analyzing the mice
Hospital Necker-Enfants Maades, Paris, France    These mice present elevated cystine levels,        for kidney, behavioral, bone and ocular
                                                 even at birth, which increase with age.            changes. In addition, in collaboration with
Our laboratory is located at Necker Hospital                                                        Dr. Minnie Sarwal at Stanford University, we
                                                 Furthermore, cystine crystals were present
in Paris, where there is a long history in                                                          are comparing the gene expression patterns
                                                 in various organs from the age of six
the Department of Pediatric Nephrology of                                                           between normal and Cystinosis mouse
                                                 months. These mice do not develop any
treating children with Cystinosis. We started                                                       tissues to identify key cellular pathways
                                                 obvious renal phenotype. In contrast, from
working on Cystinosis in 1995, when the                                                             either altered in Cystinosis or, on the
                                                 6– 8 months of age, they show decreased
chromosomal localization of the gene on                                                             contrary, involved in the protection of the
                                                 activity and osteoporosis, as well as ocular
chromosome 17 had just been found by                                                                cells. This could explain the lesser severity
                                                 lesions similar to those in patients, making
the International Cystinosis Consortium. We                                                         of the disease in the mouse model, and then
                                                 them a good model to study the mechanisms
identified the gene underlying Cystinosis in                                                        be exploited in the long term to develop
                                                 of cystine accumulation and to test new
1998, in collaboration with Dr. W. van’t Hoff                                                       new therapeutic strategies.
                                                 treatments to decrease the cellular
and Dr. M. Town in London. We subsequently
                                                 concentration of cystine.                          Another very exciting field of our research,
showed that cystinosin, as they named the
                                                                                                    currently funded by the Cystinosis Research
Cystinosis gene product, was indeed a specific   Our current research projects are aimed at
                                                                                                    Foundation, is the study of the role of
transporter of cystine located at the membrane   understanding the exact role of cystinosin
                                                                                                    cystinosin at the cellular level. We have
of the lysosomes. We also characterized          in the cells and why its alteration leads to
                                                                                                    several reasons that make us think that
mutations in the Cystinosis gene in almost       the development of Cystinosis. We are using
                                                                                                    cystinosin might also have a role in cystine
150 children from all over the world. This       both our animal model and cellular models
                                                                                                    transport in the lysosome. We are currently
allowed us to correlate the severity of the      we have recently developed in the lab.
                                                                                                    trying to demonstrate it using cell lines we
disease with the type of mutations and           The severity of the disease in the mouse           have generated stably expressing cystinosin
identify key regions in cystinosin. Since        varies depending on the strain of the mouse.       (normal or mutated) tagged with a
no animal model of Cystinosis existed,           Thus, we have transferred the mutation of          fluorescent marker, which renders it easily
we generated a knock-out mouse model,            the Cystinosis gene onto two pure mouse            detectable under a fluorescence microscope.

Ranjan Dohil, MD                                 absorbed into the blood stream and once            that a controlled-release cysteamine therapy
Associate Clinical Professor                     absorbed, how much is metabolized by the           would be possible. We are excited about the
Division of Pediatric Gastroenterology           liver as soon as it has been absorbed.             next phase of our study, i.e., creating the
and Hepatology, UCSD School of Medicine          In addition to testing CystagonTM, we will also    actual controlled-release therapy and testing
Pharmacokinetic evaluation of cysteamine         evaluate cystamine. Cystamine is an odorless       patients with Cystinosis. We recently received
bitartrate (CystagonTM) and cystamine            compound, which consist of two cysteamine          a commitment from Mylan Laboratories, Inc.,
                                                 molecules and the results of the study will        the current provider of CystagonTM, to provide
When CystagonTM was approved in 1994, the        help us assess if cystamine could be used as       the medication necessary for this study.
FDA did not require information about the        a pro-drug. The advantage of a pro-drug may        We appreciate their participation in helping
absorption, metabolism and distribution of       be a reduction of gastrointestinal side effects    to move this study forward.
CystagonTM (cysteamine bitartrate). While it     as well as the avoidance of the unpleasant
was initially seen as a benefit that the FDA     body odor CystagonTM causes. Cystamine is
did not require this information before          potentially a therapeutic option for the future.   CRF is pleased to report that the results
approving CystagonTM, this lack of knowledge     In the meantime, however, we are focused on        of Dr. Dohil’s absorption study will be
is now limiting the development of an            creating a new method of delivering CystagonTM.    published in the Journal of Pediatrics
improved way to administer the drug. The                                                            within the next few months. Once
                                                 Controlled-release Cysteamine Therapy.
aim of the animal study we have designed                                                            published it will be posted on our
is to evaluate how much of the drug is           Results from our initial study would suggest       web site at
Progress Report: Mitchondrial                            showed myopathic changes, with decreased
                                                                                                           Because of your generosity to the
                                                         amplitude and duration of motor unit action
Dysfunction in Cystinosis Myopathy                       potentials, and numerous polyphasic potentials.   Cystinosis Research Foundation
Doris A. Trauner, MD                                     Two patients had neurogenic changes, one
Professor, Departments of Neurosciences
                                                         in addition to myopathic changes and one          we have funded and committed
                                                         without myopathic changes.
and Pediatrics; Chief, Pediatric Neurology
                                                       D. Metabolic studies: Two-thirds of the patients
                                                                                                           nearly $1,600,000 in Cystinosis
UCSD School of Medicine                                   had low or borderline low plasma Co-enzyme       research. This makes the CRF
Aims of the study:                                        Q 10 levels. The same percentage had elevated
The specific aims are to determine whether the            blood CPK levels, suggestive of muscle damage.   the largest nonprofit funder of
                                                          All had normal muscle carnitine levels. These
myopathy associated with nephropathic Cystinosis
is the result of mitochondrial dysfunction with           findings add to the pathological data and        Cystinosis research in the world.
resultant deficiency in respiratory transport             suggest that muscle may be involved in
chain function in adolescents and adults with             Cystinosis even without evidence of clinical
                                                          symptoms, and the low Co-enzyme Q 10             Corrine Antignac, MD, PhD
Cystinosis, and to determine if a treatment
                                                          levels suggest that the muscle problem may       Hospital Necker-Enfants Maades,
regimen that provides additional co-factors for
these enzymes will improve the strength and               be the result of mitochondrial dysfunction.      Paris, France
prevent deterioration in muscle strength.              E. Exercise testing: Results of exercise testing    2005 - $78,000
                                                          on all of the patients have not been analyzed    Ranjan Dohil, MD
Progress to date:                                         yet, because the blinding in terms of placebo
                                                                                                           Associate Clinical Professor
‚ Enrollment                                              vs. mitochondrial “cocktail” has not been
                                                          broken. We will conduct those analyses at        Division of Pediatric Gastroenterology
Eleven patients (age range 16– 43 years) have                                                              and Hepatology,
                                                          the completion of the 12-month studies.
been enrolled in the study. One was unable to
                                                                                                           School of Medicine
continue because of prior participation in a           Future plans
study of cardiac function. Of the remaining ten,
                                                                                                           University of California, San Diego,
                                                       ‚ Complete 6- and 12-month testing.                 2002–2005 - $338,542
one has completed the 12-month visit, eight
have completed the six-month visits, and               ƒ Enter new patients as they are identified.        2005 - $200,000
one has completed the first visit. All patients          We would like a total of 20 patients.
                                                                                                           Thomas Jeitner, PhD
have been randomized to treatment with a               „ Analyze data and write manuscripts
                                                                                                           Research Assistant Professor,
“mitochondrial cocktail” or placebo. The code            with results of study.
has not yet been broken to determine which                                                                 Department of Biochemistry,
patients have received which regimen.                                                                      Medical College of Wisconsin
                                                       A Study of the Cognitive Domain of Executive        2004 –2005 - $92,680
ƒ Results of study to date
                                                       Functioning in Individuals with Cystinosis          Dzung H. Nguyen, PhD
A. Symptoms of myopathy: Only three of 11 patients
   complained of weakness and/or chewing and           Amy M. Spilkin, PhD                                 Assistant Project Scientist
   swallowing difficulties. No patient under the age   Principal Investigator                              Ajit Varki Laboratory, School of Medicine,
   of 25 had a history of weakness or easy fatigue.                                                        Glycobiology Research and Training Program,
B. Muscle biopsy: 11 muscle biopsies have              Angela O. Ballantyne, PhD                           University of California, San Diego
   been performed. Six had abnormal findings           Co-Investigator                                     2005 - $127,000
   on light microscopy. These included fiber           Progress to date: Over the first six months we      Amy Spilkin, PhD
   size variation, myofiber atrophy, ring fibers,      obtained Institutional Review Board approval
   endomysial fibrosis, vacuolar inclusions,
                                                                                                           Assistant Project Neuroscientist
                                                       for the study, ordered supplies, and trained
   increase in red granular staining with trichrome,                                                       Pediatric Neurology Research Group
                                                       a research assistant on the measures to be
   increase in lipid droplets with oil red O, and      administered. In addition, we have successfully
                                                                                                           University of California, San Diego
   type II fiber atrophy. One additional muscle        recruited, inducted, and tested 14 individuals      2005 - $167,865
   biopsy was not diagnosed as abnormal, but           with Cystinosis and 2 control participants.         Jess G. Thoene, MD
   there were findings suggestive of mild type II      Hence, we have made substantial progress
   fiber atrophy and increase in size of lipid                                                             Director, Hayward Genetics Center,
                                                       toward the goals of this study.
   droplets. Electron microscopy of the muscle                                                             Karen Gore Professor Pediatrics,
   was available for seven patients. Only two were     Results: On all 16 participants to date, we have    Tulane University, School of Medicine
   read as normal. Abnormal findings included          collected D-KEFS, Wechsler IQs, and the age-        2002 - $100,000
   filamentous bodies, ring fibers, occasional         appropriate executive functioning questionnaires    2003 - $105,000
   mitochondrial degeneration, and tubular             (BRIEF or FrSBe). We have also scored,
                                                                                                           2004 - $75,000
   intracytoplasmic inclusions. These abnormalities    reliability checked, and data entered all of the
                                                       test and questionnaire data collected so far.
                                                                                                           2005 - $200,000
   were present even in patients who had no
   complaints of muscle weakness, and who              Since we are in the first quarter of this study,    Doris A. Trauner, MD
   had good strength on muscle testing. Thus,          we have not yet analyzed any of the data.           Professor, Department of Neurosciences,
   myopathic changes in the muscle can be found        Future plans: In the next six months, we plan to    University of California, San Diego
   even in asymptomatic patients with Cystinosis.      continue to recruit, induct and test individuals    2004 - $97,300
C. EMG/NCV: We have received reports of six EMG        with Cystinosis and matched control participants,
   and NCV studies to date. Two patients had           as we work towards the projected participants       As research updates become available they
   normal studies (20 and 21.5 years old). Three       (i.e., 30 with Cystinosis, 30 controls.)            will be posted at
  $772,986                                                The Power of a Wish
  in Research Funded through
                                                          We are excited to announce that Kevin Sharp will be the guest speaker
  2005 Natalie’s Wish Event                               at our Fifth Annual Natalie’s Wish Fundraiser on Thursday, June 1, 2006
                                                          at the Balboa Bay Club in Newport Beach, California.
                                                          Kevin Sharp, award-winning vocalist, entertainer and cancer survivor, will inspire us all as
Corinne Antignac, MD, PhD                                 he shares his experiences and the lessons he learned while battling cancer. The foundations
Hospital Necker-Enfants Maades, Paris, France
                                                          of his strong beliefs are never giving in and never giving up. Kevin knows the most powerful
“Characterizations of Cystinosin
                                                          gift we have in this life is – HOPE.
Intracellular Trafficking”
$78,000 – 1-year study                                    As a teenager, Kevin was a gifted athlete who excelled in several sports. He began experiencing
                                                          fatigue and unexplained pain in his leg, which was later diagnosed as a rare form of bone
Ranjan Dohil, MD
University of California, San Diego,                      cancer (Ewings Sarcoma) that had spread to his lungs.
“Clinical Studies of Cystinosis:                          As a senior in high school, Kevin was told that his chance of survival was slim. Uncertain if he
Attempts to Improve Treatment”                            would live six months, Kevin was introduced to the Make A Wish Foundation, which grants wishes
$200,000 – 2-year study                                   to children with life-threatening illnesses. They honored his wish of meeting producer/
Dzung H. Nguyen, PhD                                      performer David Foster, whose friendship sustained Kevin through two grueling years
University of California, San Diego                       of chemotherapy, experimental drugs and radiation treatments. It also opened
“Development of a Rapid Method of                         a door for Kevin to pursue his goal to become a successful country artist.
Neutrophil/Monocyte Isolation of the
                                                          To everyone’s surprise, Kevin went into remission in 1991. He has never
Diagnosis and Monitoring of Cystinosis”
                                                          forgotten the generosity of the Make A Wish Foundation, and currently
$127,121 – 2-year study
                                                          acts as a national spokesperson for the group. He was also honored with
Jess G. Thoene, MD                                        the prestigious Wishgranter of the Year Award, which singled him out
(On hold due to hurricane Katrina,                        for the devotion, dedication and inspiration he provides the children.
research lab destroyed)                                   To date, Kevin is the only “wish child” to become the wish request of other
Tulane University, School of Medicine                     wish kids, making his wish experience come full circle.
“Lysosomal Cystine Enhanced Apoptosis in
Cultured Human Mesenchymal Stem Cells”                    A platinum debut album, Measure Of A Man; scores of award nominations;
$200,000 – 2-year study                                   and a string of chart-topping hits including She’s Sure Taking It Well, If You
                                                          Love Somebody, and the highly acclaimed Nobody Knows – which held the
Amy Spilkin, PhD
                                                          top spot on Billboard’s Country Singles Chart for an astonishing four weeks –
University of California, San Diego
“A Study of the Cognitive Domain of Executive             mark his debut as the best by a new male country singer in five years.
Functioning in Individuals with Cystinosis”               Kevin is a living example of the power of a wish and we are blessed to have him
$167,865 – 2-year study                                   share his music and his story with us at Natalie’s Wish on June 1, 2006.

                     I wish I may, I wish I might, have the wish I wish tonight.

                                                                                                                                        PRESORTED STANDARD
  CYSTINOSIS RESEARCH FOUNDATION                                                                                                            U.S. POSTAGE
  18802 Bardeen, Irvine, California 92612-1521                                                                                                PAID
  949.809.2400 •                                                                                                       SANTA ANA, CA
                                                                                                                                            PERMIT NO. 3
  501(c)3 Federal Tax ID #32-0067668

          Save the Date                               y
                      THE FIFTH ANNUAL                y
           “NATALIE’S WISH” FUNDRAISER                y

                THURSDAY, JUNE 1, 2006                y
               Newport Beach California

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