Drug aimed at Huntington's eases chorea, the disease's hallmark feature

A drug widely available in Europe and Canada but not the United States dramatically eases one of the most disabling symptoms of Huntingtons disease, involuntary writhing movements known as chorea, according to a study in the Feb. 14 issue of the journal Neurology.

The medication, tetrabenazine, is currently being reviewed by the U.S. Food and Drug Administration. If approved, the medication would be the first authorized by the agency expressly for the treatment of Huntingtons disease, which affects about 30,000 people in the United States.

In a randomized, controlled study conducted in 84 patients at 16 sites around the nation, doctors found that the medication cut down involuntary movement on average by about 25 percent, with many patients experiencing a greater improvement. Overall, patients who received the medication were six times as likely to be considered by their doctors to have improved considerably, compared to participants who received a placebo.

Neuroleptic drugs like haloperidol (Haldol) are currently in widespread use in the United States to suppress chorea, but the effect of these drugs on chorea has never been studied in a systematic way, and they have a number of troublesome side effects, such as blunting of personality, loss of voluntary movement, and hindering balance. Our study showed that tetrabenazine, when appropriately dosed, can decrease chorea without causing those side effects, said Frederick J. Marshall, M.D., a neurologist at the University of Rochester Medical Center who led the study conducted by the Huntington Study Group. The study was funded by Prestwick Pharmaceuticals of Washington, D.C., the company that owns the rights to develop and sell the medication in the United States.

Tetrabenazine was originally developed in the 1950s to treat psychosis, but was quickly pushed aside by more effective medications. But doctors in the United Kingdom found it to be effective to treat the excessive involuntar

Contact: Tom Rickey
University of Rochester Medical Center

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