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.
Dr. Kathleen M. Shannon, neurologist and Huntington's disease specialist at the Huntington's Disease Society of America Center of Excellence at Rush University Medical Center, led the Rush study. Rush was one of 16 sites to participate in the randomized, controlled study which involved 84 patients.
Tetrabenazin is available in Europe and Canada but not the United States, but 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 Huntington's disease, which affects about 30,000 people in the United States.
"Huntington's disease is an inherited brain disorder that causes patients to experience uncontrollable jerky movements (chorea), as well as changes in personality, behavior, thinking and memory. There are no FDA-approved treatments for the chorea. Anti-psychotic drugs like haloperidol (Haldol) are commonly used to suppress chorea, but they cause many different side effects. This study shows that tetrabenazine can decrease chorea, and the drug is well-tolerated by most research subjects," said Shannon.
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 involuntary movements of Huntington's, and it is approved for use in several nations. In the United States, tetrabenazine is designated as an "orphan drug" by the F
Contact: Mary Ann Schultz
Rush University Medical Center