Gene Therapy Shows Promise in Treatment of Parkinson’s Disease

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Results from a clinical trial have shown that cutting-edge gene therapy on Parkinson’s disease patients significantly improved the tremors, rigidity and other motor skill problems that are associated with the disease, US News and World Report reported.

The study, involving 45 patients with moderate to advanced Parkinson’s disease, randomly assigned half of the patients to receive the gene therapy and the other half to undergo a mock procedure designed to mimic the therapy.

The therapy was done with local anesthesia and used a harmless, inactive virus to deliver a glutamic acid decarboxylase (GAD) gene into the part of the brain that regulates motor function. The GAD gene was meant to prompt the production of a chemical that improves motor control, and therefore re-establish the normal chemical balance that becomes dysfunctional as Parkinson’s disease progresses.

According to the study authors, the 22 patients whose brains were infused with the GAD gene experienced “clinically meaningful improvements” of their symptoms within six months of surgery – a 23.1 percent improvement versus a 12.7 percent improvement in placebo patients on a scale used to assess motor function in Parkinson’s patients.  Furthermore, the treatment did not appear to have any serious, adverse effects.

“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.

Parkinson’s disease affects one million aging Americans and is the second most common neurodegenerative disease after Alzheimer’s, according to the National Parkinson Foundation.

The study is was published in The Lancet Neurology.

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