Huntington's Disease Society of America Researchers Uncover Possible Treatment for Huntington's Disease

Huntington's Disease Society of America
Monday, 22 October 2001

New York, NY, October 22, 2001 – In an exciting research article published on October 18 in the journal Nature, Drs. Leslie Thompson and Joan Steffan, along with a team of scientists at the University of California at Irvine, made headway in halting and preventing the neurodegeneration seen in Huntington's Disease.

Dr. Thompson, an HDSA Coalition for the Cure investigator, led the team in a project that showed how chemicals known as histone deacetylase (HDAC) inhibitors used in a fruit fly model of Huntington's Disease can prevent cell death caused by the HD mutated gene.

The team's premise is that the mutant huntingtin protein disrupts the action of key enzymes which are called acetyltransferases (ATs), thus destroying the delicate balance normally seen in cells that do not carry the HD gene and protein. To restore this balance, the team added chemicals that inhibited the HDAC, thus resulting in the prevention of nerve damage caused by HD.

This research was achieved using drugs that are currently in human cancer clinical trials and have already been approved by the Food and Drug Administration. This would result in a swifter move to testing in HD mouse models and then into HD clinical trials.

Huntington's Disease is an inherited, degenerative brain disorder that results in the progressive loss of control of both the mind and the body. Each child of an affected parent has a 50% chance of inheriting the disease. Presently, there is no effective treatment or cure for this deadly illness that affects 30,000 Americans and places another 200,000 at-risk.

Since 1996, the Huntington's Disease Society of America has increased its annual commitment to HD research from $183,000 to more than $4 million in the coming year.

These vital funds are what made this research breakthrough possible, as well as our most recent discoveries by HDSA Coalition for the Cure investigators Christopher Ross, M.D., Ph.D. (Science, March 23, 2001) and Elena Cattaneo, Ph.D. (Science, June 14, 2001).

For more information, or to contact Huntington's Disease Society of America, see their website at: www.hdsa.org