Transplant Drug Aids Huntington's

From the Summer 2004 issue of Hopes & Dreams, newsletter of the Illinois Chapter, Huntington's Disease Society of America.

Scientists in the UK have found a drug which appears to slow the progress of the debilitating condition Huntington's Disease, which currently has no cure. Animal tests showed that rapamycin also delays the onset of the disease. The drug is already used in humans to prevent organ rejection after transplants. Huntington's Disease groups hailed the research as a significant advance.

Balance risks with benefits The research found that rapamycin can reduce the levels of a toxic protein causing Huntington's. It does this by speeding up the break-down of the protein in cells. Rapamycin is designed for long-term use, which is obviously crucial for someone who has this disorder. It is not without side effects, but you could argue that you'd be balancing side effect risk with the potential benefits. More research would need to be carried out before the drug could be used to treat Huntington's in humans, but studies would be done "as fast as possible".

Researchers noted that the fact it was possible to test someone to assess their risk of developing Huntington's meant it would be easy to target the drug treatment at those who would benefit from it. At the moment, the drugs that are used are to control each of the individual symptoms, not the disease. This research means it might be possible to delay the onset of symptoms. It could give people a lot more life.

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Created: Aug. 11, 2004
Last updated: Nov. 15, 2010