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Gene Therapy for Parkinsons Disease

According to the first scientific review of a dozen patients who have received the treatment over the last three years ,a novel gene therapy technique is safe and may be effective at staving off worsening symptoms of Parkinson's disease.

The patients, half of whom live on Long Island, are in advanced stages of the illness and were no longer responding to medicines when they signed on for the experimental therapy. The study was conducted by Andrew Feigin, MD, director of Neuroscience Experimental Therapeutics at The Feinstein Institute for Medical Research and his colleagues in collaboration with Parkinsons scientists at New York Presbyterian Hospital-Weill Cornell Medical Center in Manhattan.

One woman and 11 men received a surgical infusion of fluid containing a viral vector and genes for a protein called GAD, glutamic acid decarboxylase. This enzyme is critical in controlling a neurotransmitter called GABA. In Parkinsons, GABA is reduced in an area of the brain called the subthalamic nucleus.

This region is working on overdrive in the disease process and GABA is an inhibitory transmitter and is important in trying to calm this hyper-reactive circuit.

The gene therapy would be used to reduce symptoms and not alter the underlying disease process. Finding novel therapies are key as many Parkinsons patients stop develop complications after prolonged use of traditional medicines.

The Feinsteins David Eidelberg, MD, took brain scans before, during and after the treatment and the scans show that the brain is re-working these abnormal circuits. Dr. Feigin said that patients had about a 27 percent improvement in symptoms, although the study was an open label design.

The scientists are now designing a double-blind placebo controlled trial that would enroll far more patients in an attempt to see whether the gene therapy is effective in reducing symptoms.

The patients' scans showed a quieting of thes
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