Parkinson’s Disease Gene Therapy Breakthrough Reduces Symptoms – Advanced Condition When Patients Fail To Respond Medicines

Parkinson’s Disease Gene Therapy Breakthrough Reduces Symptoms – Advanced Condition When Patients Fail To Respond Medicines

Dr. Andrew Feigin

(Best Syndication) Scientists in New York say that a special gene therapy treatment may help advanced Parkinson’s disease sufferers who do not or stopped responding to current medicines.

Gene therapy may be a better way to treat Parkinson’s disease. “Gene therapy could be a more natural way to treat the disease,” said Dr. Andrew Feigin, MD,director of Neuroscience Experimental Therapeutics at The Feinstein Institute for Medical Research. He collaborated with Parkinson’s disease researchers at the New York Presbyterian Hospital-Weill Cornell Medical Center in Manhattan.

The study involved one woman and 11 men who were in advanced stages of the illness and who were no longer responding to medications. The patients were given an infusion of fluid containing a viral vector and genes for a protein called GAD, glutamic acid decarboxylase. The Feinstein’s David Eidelberg, MD, took brain scans before, during and after the treatment.

The enzyme is critical in controlling a neurotransmitter called GABA (Gamma-aminobutyric acid). In Parkinson’s patietnts, 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.

Since Parkinson’s patients have worsening symptoms on one side of the body, the researchers injected the genes into one side of the brain. The brain scans showed a quieting of the areas on the side of the brain where the genes were infused.

Although this strategy is designed to reduce symptoms and not alter the underlying disease process, the researchers found that since they began in 2003, some patients continue to show improvement. “This important study shows that gene therapy can be performed safely and may benefits patients,” Feignin says.

The scientists are planning 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 complete study is published in the latest issue of Lancet.

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By Marsha Quinn
Best Syndicaiton News Health Writer



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