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Gene Therapy May Help Reverse Parkinson's Symptoms
Date:3/17/2011

By Maureen Salamon
HealthDay Reporter

THURSDAY, March 17 (HealthDay News) -- Cutting-edge gene therapy on Parkinson's disease patients significantly improved the tremors, rigidity and other motor skill problems that are hallmarks of the illness, a small new study reports.

The phase 2 study enrolled 45 patients with moderate to advanced Parkinson's disease, with half randomly assigned to receive the gene therapy and half assigned to undergo a "sham" surgery, a mock procedure designed to mimic the experimental approach.

Of the 22 patients who were infused directly into the brain with the glutamic acid decarboxylase (GAD) gene -- which prompts the production of a chemical known as GABA that improves motor control -- half experienced "clinically meaningful improvements" of their symptoms within six months of surgery, the study authors said.

Although several open-label trials on gene therapy have shown promise in treating neurologic disease, the researchers noted that this is the first of its type to be confirmed in a follow-up randomized double-blind trial, a study in which neither the investigators nor the patients knew which patients were receiving the real or sham treatment.

"It's a completely novel therapy -- unlike anything that's currently offered," said co-investigator Dr. Andrew Feigin, an associate investigator at the Center for Neurosciences at Feinstein Institute for Medical Research in Manhasset, N.Y.

"I think we expected to see this effect, but you never know," Feigin added. "I was gratified."

The study is published online March 17 in The Lancet Neurology.

Parkinson's disease affects about one million Americans, most of them over age 50, and is the second most common neurodegenerative disease after Alzheimer's, according to the National Parkinson Foundation.

In addition to confirming results of earlier clinical trials, the treatment didn't have any serious adverse ef
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