Doctors ordinarily use drugs to control the individual symptoms of HD, but the drug rapamycin may delay the onset and progression of HD altogether, as suggested by recent preliminary animal test results from Cambridge University researchers. Rapamycin is currently used to prevent graft-versus-host disease (GVHD) in people who have received organ transplants. The drug, which reduces the levels of the toxic (mutated) huntingtin protein, is designed for long-term use, which would benefit HD sufferers due to the lengthy course of the disease. Rapamycin increases the rate at which mutant huntingtin breaks down in the nerve cells of the brain.
The principal researcher acknowledges that, like any other drug, rapamycin has potential side effects. However, more research will be done as soon as possible to make sure that it is safe for humans. He also said that since people can opt to have a genetic test to evaluate their risk of developing HD (although many people choose not to be tested), it would be easy to target rapamycin treatment to those who would benefit from it. Perhaps when rapamycin is deemed safe and becomes available, more people may be willing to undergo the genetic test. For more information about genetic testing for HD, click here.
To read the full articles, please click here and here.
Last Modified: 05/22/2009
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