First drug for Huntington's disease

The first effective treatment for the symptoms of Huntington's disease has been approved by the U.S. Food and Drug Administration. The drug won approval after a study by Baylor College of Medicine and the Huntington Study Group, an international group of physician scientists, showed the drug's effectiveness.

Huntington's disease is a rare, inherited neurological disorder affecting about 30,000 people in the United States. Another 200,000 are at risk of developing the condition.

One symptom of Huntington's disease is abnormal involuntary movement, called chorea, which is thought to be caused by overactivity of the neurotransmitter dopamine. The drug, tetrabenazine (which will be marketed as Xenazine), eases these movements for patients.

"Tetrabenazine works by depleting dopamine levels in the brain," said Joseph Jankovic, M.D., Professor of Neurology and Director of the Parkinson Disease Center and Movement Disorder Clinic at BCM. "In 1979, I was granted special permission from the FDA to treat patients with the drug. Since that time, I've treated thousands of people with tetrabenazine and have seen amazing results that translated into marked improvement in the quality of life."