Hereditary Diseases: Promising New Research

[Guest guest]

Hereditary Diseases: Promising New Research

http://www.medicalnewstoday.com/medicalnews.php?newsid=50326

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 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cutting-edge and unpublished data to advance the search for a cure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. , 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

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, s 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.

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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.