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THE markerHuntington’s Disease Society of America HDSA Research Update 2001 progress hd living in research kids at-risk care a message from n the 1960’s, activist and entertainer In our Nation’s Capital, HDSA advocacy Truly, the answers Woody Guthrie was well known efforts have been unflagging. As a result of we find for Ithroughout the world, but the disease our awareness building efforts, HDSA was Huntington’s that ended his life was not. One of the invited by Connecticut Senator Disease may well greatest challenges that faced Marjorie Christopher Dodd in March to testify serve as the key to Guthrie when she founded the before his Senate Biotechnology caucus. effective therapies organization that was to become the In May, HDSA was invited to partner with or even cures for Huntington’s Disease Society of America the National Institute of Neurological all neuro- was the fact that HD was a “family” Disorders and Stroke (NINDS) in a pilot degenerative secret, misunderstood and often project that was reported in the last issue of and/or hereditary misdiagnosed. Educating healthcare Toward a Cure. In June, HDSA appeared at disorders. professionals, elected officials and the a Health Policy Briefing, hosted by the The HDSA Therapeutics Initiative will general public about Huntington’s National Health Council, along with bring the research, conducted by our Disease, and the challenges faced by several other voluntary health agencies, HDSA Coalition for the Cure those who live with it, has always been to bring issues of concern to the HD investigators and Grant and Fellowship an integral part of HDSA’s mission. community to congressional staffers. Our recipients, to clinical trials more rapidly. dedication to increasing awareness about I am very pleased to report to you the But to continue these intensive research this deadly disease, and sharing our message success that HDSA has achieved this efforts, HDSA must pledge even more to of hope through research with our elected past year in increasing awareness on all fund the needs of our investigators. To officials, remains a priority as we move levels through a new public awareness that end, HDSA launched phase two of forward in this congressional session. Their program. Many of you have seen the eye Generation 2000: Fulfilling the Promise in understanding of the multiple issues that catching ads for HD and HDSA that an effort to raise $25 million for HD face HD families as they battle this have appeared in major national research over the next five years. The progressively degenerative disease can lead magazines, including Business Week, answers are close at hand. I ask that you to increased support for HD research and Newsweek, Bon Appetit and dozens of consider making a generous donation to new legislation on issues of importance to others, thanks to the generosity of an this research campaign today. Every dollar our HD families and friends. in-kind donation program with a major contributed will be matched by the media placement firm. Public Service Through these advocacy efforts and the Research Matching Gifts Challenge Fund. Announcements (PSAs) have aired in recent amazing advances in HDSA Won’t you please join with me in helping the top 25 radio markets in the US and sponsored research, scientists worldwide to make this the last generation with HD? represent a gift of more than $306,000 of now view HD as a “model” for other Sincerely, free radio time. Millions of people who neurodegenerative and hereditary diseases. may never have heard of HD now have a HD is now being linked to Parkinson’s greater understanding of the devastation disease and ALS (Amyotrophic lateral it causes and our optimism that a cure is sclerosis) to advance awareness in ads Don King, Ph.D. on the way. created specifically by those organizations. The Marker is an official publication of the The Marker, a periodical of the Huntington’s The Society is a member of the National Health Huntington’s Disease Society of America, Inc. Disease Society of America, Inc., is published Council, the National Foundation for Brain 158 West 29th Street, 7th Floor, New York, NY twice annually. Its purpose is to provide Research, the International Huntington 10001-5300. (212) 242-1968 information and opinion and to relay items of Organization, the National Organization for Rare Donald A. King, Ph.D. interest to individuals with Huntington’s Disease Disorders, the National Voluntary Health Chair of the Board and their families, health care professionals, and Agencies, the Alliance of Genetic Support Groups interested friends and supporters. and the Independent Sector. Barbara T. Boyle National Executive Director/CEO The appearance of advertising, or the mention of The Huntington’s Disease Society of America commercial products available for sale in articles meets all nine standards of the National Charities Debra Lovecky published in this publication, is not an HDSA, Inc. Information Bureau. Director of Communications/Editor guarantee or endorsement of the product or the ©2001 Huntington’s Disease Society of America Karen Tarapata claims made for the product by the manufacturer. Editor Statements and opinions expressed in articles are Byne Graphics not necessarily those of HDSA, Inc. About the front cover: Design Different views of brain imaging. HDSA, Inc. is a national not-for-profit Corri Jones organization founded in 1986 to help individuals Art Director with Huntington’s Disease and their families. a message from s we enter this season of giving, In May, the National Institute of on that let us pause to reflect on all that Neurological Disorders and Stroke cataclysmic Awe have been able to accomplish (NINDS) invited HDSA and ALSA Tuesday by through the generosity and dedication of (Amyotrophic Lateral Sclerosis remembering our HD families, friends and corporate Association) to partner with them on a them with supporters. $1.2 million pilot project aimed at gratitude, living developing assays that would be tested each day with We began 2001 auspiciously with the against 1,000 FDA-approved compounds hope, and announcement that phase one of for possible therapies. This historic continuing my Generation 2000 - Making This the Last collaboration coincided strategically with work until it is Generation with HD had raised more than HDSA’s invitation for Coalition for the done - until a cure is found for $2.7 million for HDSA research Cure researchers to advance projects Huntington’s Disease. initiatives. This more than surpassed our through the new HDSA Therapeutics original goal of $500,000 and even our Today, as we enter the season of Initiative that will use high-throughput dream of raising $2 million. Building on thanksgiving and renewal, I invite you to screens to assess potential therapeutic the incredible momentum of phase one, join with me to give a gift of hope to applications. HDSA launched phase two - Fulfilling the your family, friends and associates that Promise - in January 2001 with a goal to In July, at the 16th Annual HDSA will last for years to come. The raise $25 million for HDSA research Convention, the much anticipated Christopher Radko Company has created programs and initiatives over the next five Journal of Hope was presented to those in a limited number of very special years. Spurred on by the success of attendance with a direct mail follow up to mementos that symbolize our hope for a HDSA’s original Matching Gift Fund, those who contributed but were unable to future free of Huntington’s Disease. Every several HD families came forward to attend the convention. More than 350 dollar raised through the Gift of Hope create a new matching fund for Fulfilling inspiring and oftentimes poignant campaign will go to HDSA’s research the Promise that would challenge our messages accompanied the listing of the programs and initiatives. Personal friends and families to increase their level more than 2,400 individuals, families and messages will accompany keepsakes that of support in order to attain our $25 corporate friends who made phase one of have been purchased for family members, million goal. This Research Matching Gifts Generation 2000 an astounding success. friends and associates. Please take a few minutes to read about our partnership Challenge Fund currently stands at $2.5 On September 11th our nation faced its with the Christopher Radko Company million and all gifts made to Generation gravest threat to our freedom yet known. on page 33 and please consider giving a 2000: Fulfilling the Promise will be matched On that sunny Tuesday morning, forces Gift of Hope this year. We have come so dollar for dollar. that do not believe in the very freedom far in the last five years. We cannot give As part of the annual leadership conference that we take for granted attacked us. up now. Together, we can make this the held in March, HDSA leaders met with Many innocent people were killed and a last generation with HD. their elected representatives in Washington, nation mourned the loss of those lives DC for a “Day on the Hill.” Advocacy and a way of life that we will never know Sincerely, training was conducted by McDermott, again. In the intervening months, each of Will and Emery with additional follow up us has picked up the pieces of our lives training for “home office” visits and and, as our President asked, done our best Barbara T. Boyle continued contact with their elected to return to a normal routine. I, for one, representatives. plan to honor our fellow Americans lost table of contents Our special thanks Fighting Back Against HD ................2 Youth and Juvenile HD ....................24 to HDSA Coalition for the Cure ............4 Chapters ..........................................27 AstraZeneca HSG Update ......................................9 Centers of Excellence ......................28 for the Grants and Fellowships ..................10 HDSA Convention 2001 ..................30 Community Service Grant Caregiver’s Link................................20 HDSA Advocacy ..............................32 that has made this Living At-Risk ..................................23 Giving Opportunities ......................34 publication possible Fighting Back Against HD by Christopher Ross, M.D.