New Huntington's Disease Research Shows Promise

 New York, NY, February 7, 2002 - In the February issue of Nature Medicine, HD researcher, Marcela Karpuj in Larry Steinman’s laboratory at Stanford University Medical Center reported on a potential treatment for Huntington's Disease. Using mouse models of the disease, researchers were able to reduce the uncontrollable movements that characterize HD and extend the lives of the mice.

 This was accomplished by treating mice, exhibiting the symptoms of HD, with cystamine, a compound, which has a number of different biochemical effects. The treated mice showed signs of improved gait and less weight loss, abnormal movements lessened, and life span was increased by 11%. The huntingtin aggregates remained unchanged, which surprised the researchers. They had theorized that cystamine would inhibit the enzyme transglutaminase, which forms a chemical link within huntingtin aggregates, and thus provide a protective response. However, the aggregations were the same after treatment.

 The researchers then began to examine the brains of mice treated with cystamine compared to those not treated with the compound. They found that the mice treated with cystamine had elevated levels of three neuroprotective proteins, suggesting some of the mechanisms by which the brain tries to protect itself. These same proteins were found at increased levels in the brains of human HD patients.

 Research conducted by HDSA Coalition for the Cure investigator, Steven Hersch, M.D., Ph.D. and grant recipient Robert Ferrante, Ph.D., has found that cystamine has an even greater therapeutic effect on HD mouse models when treatment is started earlier. Dr. Hersch and Dr. Ferrante, at Massachusetts General Hospital and Boston University have seen increased life spans by up to 19% while also dramatically reducing aggregate formation. Their research was presented at the Society of Neuroscience, which met last November.

 Cystamine is not a drug designed for human use, however related compounds that might be tested in human clinical trials are already being examined by both the HDSA and the Huntington Study Group.

 Neuroprotective compounds may be just one of the many ways that patients affected by HD may be treated in the future. There are many avenues of research that remain promising and any one of them may lead the way to an effective therapy and eventually a cure for Huntington's Disease.