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First Gene Therapy Study for Parkinson's

August 19, 2003, The New York Times reported that a patient at NewYork Weill Cornell Medical Center became the first person to undergo gene therapy for Parkinson's. The ambitious experiment involved the infusion deep into the brain of 3.5 billion viral particles, each bearing a copy of a human gene meant to help relieve the tremors, shuffling gait and other abnormal movements caused by Parkinson's disease.

The procedure was performed by Dr. Michael Kaplitt, an assistant professor at Weill-Cornell Medical Center. The U.S. Food and Drug Administration approved this experimental procedure for 12 patients with severe Parkinson's. The experiment is a Phase 1 trial, meaning that its main goal is to determine safety, not efficacy. Inevitably, researchers and their subjects will also be looking for signs that the treatment works. That should become clear within three months, Dr. Kaplitt said.

It is known that patients with Parkinson's disease have excessive electrical activity in a deep part of the brain called the subthalamic nucleus. Dr. Kaplitt and his team proposed that transferring a gene for the enzyme glutamic acid decarboxylase, or GAD, could help Parkinson's disease by shutting down the overactive circuit. As reported in the October 2002 edition of the journal Science, when the gene transfer technique was performed in rats, the GAD enzyme was successfully produced in brain cells, and the rats were protected from becoming parkinsonian.

The gene-transfer experiment has drawn criticism from experts in Parkinson's disease who feel that there has not been enough preliminary evidence to justify injecting a virus into the human brain. The experiment has not been done in primates, and experts are concerned that the virus could multiply and spread beyond its intended target, or churn out huge quantities of unwanted enzyme.

Robin Elliott, Executive Director of the Parkinson's Disease Foundation commented:
"Although we have no involvement with this study, we believe that gene therapy is a promising and legitimate area for exploration in the quest for easing the burden of people with Parkinson's disease. Dr. Kaplitt's approach is innovative and creative and conducted under the safety mechanisms established by the U.S. Food and Drug Administration to protect patients."

For the full story see the "Science News" section of the PDF website: www.pdf.org.