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Sunday, March 20, 2011
Gene therapy may help reverse Parkinson's symptoms
From USA Today: 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 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.
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