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Entries in Huntington's Disease (1)

Tuesday
Nov082011

Promising New Treatment for Huntington's Disease

Brand X Pictures/Thinkstock(MADRID) -- There is no cure and there are few treatments available for patients with Huntington's disease -- a devastating degenerative condition that breaks down nerve cells in the brains -- but a new study published in the Lancet Neurology found that a drug known as pridopidine seems well-tolerated and efficient in helping patients control involuntary movements.

"The study shows modest improvement of a variety of motor deficits without relevant side effects in patients with Huntington's disease," said Dr. Justo Garcia de Yebenes, lead author of the study, which was based in Madrid.  "For the moment these results contribute to the symptomatic treatment of a variety of motor deficits in patients with this disease."

The disease is a debilitating genetic disorder that affects about 30,000 Americans.  The condition causes involuntary movements, coordination and speech and swallowing problems, along with dementia.  Children of parents with a single mutated gene have a 50 percent chance of developing it.

Onset of the disease typically begins around 40 years old, and patients usually die 15 to 20 years after symptoms surface, according to the Huntington 's Disease Society of America.

The study drug under investigation, pridopidine, works by stabilizing dopamine in the central nervous system, researchers said, particularly in areas of the brain that control movement and coordination.  There is only one other drug, known as tetrabenazine, which treats involuntary movements in patients.

"My general impression is that the results were modest in magnitude, but the safety and tolerability was excellent so the drug has definite promise," Dr. Karl Kieburtz, professor of neurology at University of Rochester Medical Center, said in an email.  "The studies completed to date will likely need confirmation before approval by regulators, but such an addition to the treatments for HD (very few indeed) would be most welcome by physicians, patients and families."

The phase three trial analyzed the safety and efficacy of the treatment among 437 HD patients from eight countries, in which study participants were randomly assigned to take the drug or the placebo for more than six months.  The pridopidine improved eye and hand movements, along with balance, when compared with patients taking the placebo.

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ABC News Radio