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Breaking News: Scientists gathered to report
promising innovative strategies and potential future therapies for
Huntington's disease and related disorders.
Hereditary Disease Foundation Symposium HD 2006: Changes, Advances
and Good News (CAG)n August 10, 2006 to August 14, 2006 Cambridge,
Massachusetts
Cambridge, MA, August 21, 2006 - An
international group of stellar scientists gathered together for four
days of intense discussion, debate and designing future therapies
for Huntington's disease and related disorders. From August 10 - 14,
2006 the Hereditary Disease Foundation convened HD 2006: Changes,
Advances and Good News (CAG)n in Cambridge, Massachusetts.
Almost 400 scientists met together to share their latest
discoveries. "The synergy, innovation and enthusiastic collaboration
fostered by this gathering of dedicated scientists brings out the
best in all of us and will hopefully lead us to a lifesaving cure
for Huntington's disease!" stated Nancy S. Wexler, Ph.D., President
of the Hereditary Disease Foundation and Higgins Professor of
Neuropsychology in the Departments of Neurology and Psychiatry of
the College of Physicians and Surgeons at Columbia University, in
her opening remarks. "We are gratified that so many excellent
scientists from around the world participated and presented their
latest, cutting-edge and unpublished data to advance the search for
a cure for Huntington's disease," said Carl D. Johnson, Ph.D.,
Executive Director for Science of the Hereditary Disease Foundation.
Topics covered:
* Unraveling the cause of Huntington's disease
* Novel therapeutic strategies
* Advances in RNA interference therapy
* Discovering new drugs
* Clinical trials
Participants described the meeting as "the definitive Huntington's
disease meeting in the world for this year."
Highlights of the meeting included the following:
An extremely promising innovative strategy presented involves
tackling the problem at its very source. DNA in the gene causing
Huntington's disease expands too much. This abnormal DNA is copied
faithfully into RNA. The RNA then makes a protein which is also
abnormal. This abnormal protein wreaks havoc and eventually is the
cause of cellular disability and death.
Extraordinary new techniques target the RNA and prevent it from ever
making the abnormal protein at all. This strategy is virtually a
cure not only for Huntington's disease but also for hereditary forms
of Alzheimer's disease, Parkinson's disease, Lou Gehrig's disease,
other genetic diseases and even cancer. The meeting presented
fabulous advances in the strategies which are called RNA
interference. All of these breakthroughs are currently curing
Huntington's disease in mice. They are also being tested for safety
in monkeys. Participants grappled with the challenges of rapidly
moving these discoveries to people.
Participants also heard the very promising results of cell
transplant therapy. People with Huntington's disease in France
received cellular transplants into their brains, which then grew and
made connections. The initial pilot phases of this research were so
encouraging, even after a follow-up of 8 years, that the research
has expanded to include 60 or more HD patients in Europe. These
clinical trials are being conducted in such a rigorous fashion that
they will enable us to learn a great deal about the future of cell
replacement therapy.
Participants also learned about significant advances in the
development of new stem cell lines. These lines of neurons - or
brain cells - could also benefit patients and could potentially be
used as a reliable and renewable source of tissue for cell
replacement therapy. Interesting and promising pilot research
findings were presented using high dose creatine as a therapy for HD
patients. At the mouse level, new intriguing findings were reported
concerning BDNF, brain-derived neurotrophic factor. BDNF is a kind
of support and survival factor without which brain cells can die -
like a kind of food for brain cells. When BDNF was administered to
mice with Huntington's disease, their lot improved greatly.
Participants were introduced to a new initiative being undertaken by
the Hereditary Disease Foundation in collaboration with the European
Huntington's Disease Network (EHDN). This initiative involves the
development of new guidelines for the care of people with HD, based
on the evidence of carefully controlled clinical trials - whenever
possible. A follow-up workshop on Monday, August 14, 2006, to
develop the guidelines' procedures was hosted by the HDF, EHDN and
Sir Michael D. Rawlins, Chair of the United Kingdom's National
Institute of Health and Clinical Excellence (NICE).
The Hereditary Disease Foundation's biennial meeting once again
brings together an international cadre of junior and senior
scientists who comprise the top experts and drivers of the field. It
draws both longtime HD researchers and investigators just entering
the field. The meeting is a magnet for all the key players.
Collaborations are formed which catalyze the research of the future.
Researchers from universities and government organizations from
around the world attended, including large delegations from Harvard
Medical School and Massachusetts General Hospital, Massachusetts
Institute of Technology, Johns Hopkins University, the Universities
of California at Los Angeles, San Francisco and Irvine and the
University of British Columbia as well as smaller groups from nearly
50 other university and research centers around the world. In
addition there were participants from biotech and pharmaceutical
companies including, Novartis, Merck, Alnylam, Medtronics, Cambridge
Laboratories, Trophos, EnVivo, CombinatoRx, Metabolon, Psychogenics,
BioFocus, Cambria and Addgene.
The Hereditary Disease Foundation gratefully received support for
the meeting from Alnylam Pharmaceuticals, Inc., Cambridge
Laboratories Ltd, EnVivo Pharmaceuticals, Inc., Fisher Scientific
International, Inc., Genzyme Corporation, Merck & Co., Inc.,
Novartis Institutes for BioMedical Research, Inc., Pfizer Inc.,
Prestwick Pharmaceuticals, Inc., Sirna Therapeutics, Inc. and Vertex
Pharmaceuticals, Inc.
Huntington's disease is a fatal neurological illness causing
involuntary movements, severe emotional disturbance and cognitive
decline. Huntington's disease usually strikes in mid-life, in the
thirties or forties, although it can also attack children and the
elderly. There is no treatment to halt the inexorable progression,
which leads to death after ten to twenty-five years. Each child of a
parent with Huntington's disease has a 50% risk of inheriting the
illness. It they inherit the abnormal gene and live a normal life
span, the disease invariably appears. In the United States, the
prevalence of the disease is about 10 people per 100,000 people -
about 30,000 symptomatic people in all. There are another 150,000
individuals at risk. This means that approximately 100,000 people
may need therapy daily if taking a drug to prevent the disease from
ever occurring - a preferred mode of treatment.
Because Huntington's disease destroys so many different capabilities
- intellectual, physical and emotional - the insights gained from
research on this illness are relevant to the understanding of many
other diseases, including schizophrenia, manic depression,
Alzheimer's disease, Lou Gehrig's disease, Parkinson's disease and
cancer. By focusing on Huntington's disease as a model, the
Hereditary Disease Foundation targets neurological and genetic
functions relevant to a broad spectrum of disorders.
The Hereditary Disease Foundation (http://www.hdfoundation.org)
was founded in 1968 and is dedicated to finding a cure for
Huntington's disease and related disorders. The HDF's unique
combination of creating and funding innovative, strategic research
has sparked an explosion of knowledge in the scientific field.
Breakthroughs by the HDF's scientists include the landmark discovery
of the Huntington's disease marker in 1983 and the gene itself in
1993. The Hereditary Disease Foundation and its scientists also
helped to launch the Human Genome Project. Collectively, they have
created a vast, international, collaborative effort that is markedly
shortening the journey towards a cure.
The Hereditary Disease Foundation's research programs include grants
and John J. Wasmuth postdoctoral fellowships. Special awards,
selected by the Scientific Advisory Board of the Hereditary Disease
Foundation, include the Milton Wexler Postdoctoral Fellowship Award
and the Lieberman Award. A centerpiece of the Foundation is the
program of interdisciplinary Mary Jennifer Selznick workshops held
several times during the year. These small, informal, dynamic
workshops foster dialogue among researchers from a variety of
fields, who come without prepared lectures or slides to converse
across disciplinary borders.
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You can learn more about the event by reading our Workshop Report.
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