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New study shows efficacy of gene therapy for Parkinson's disease

October 11, 2002 Auckland, New Zealand and New York, NY: In a study published today in the journal Science, scientists from the University of Auckland and Weill Cornell Medical College reported on the effectiveness of a new gene therapy approach to Parkinson's Disease, and the potential for this therapy to affect the overall progression of the disease itself. Based on this study and other data, the U.S. Food and Drug Administration (FDA) has given its approval to begin testing this therapy in a small Phase I clinical trial. This will be the first time in the world that gene therapy will be used in patients with Parkinson's Disease.

The Science publication is authored by lead investigator, Dr. Matthew J. During, Professor of Molecular Medicine at the University of Auckland, first author Dr. Jia Luo, and co-investigator Dr. Michael G. Kaplitt, Director of Stereotactic and Functional Neurosurgery and Asst. Professor at Weill Cornell Medical College. Dr. During and Dr. Kaplitt are also co-principal investigators on the upcoming clinical trial of this therapy.

"We are using gene therapy to "re-set" a specific group of cells that have become overactive in an affected part of the brain, causing the impaired movement and other symptoms associated with Parkinson's Disease," said Dr. During. "We are very encouraged that in addition to the affect this therapy has on quieting symptoms, we present evidence that suggests it may arrest or delay disease progression."

People with Parkinson's Disease have a profound loss of a specific group of nerve cells deep in the brain which make dopamine, a signaling molecule. The loss of dopamine leads to a disturbance in the brain's network activity controlling movement. In the center of this network is a region called the subthalamic nucleus (STN), which in Parkinson's Disease is extremely overactive, and if silenced leads to a dramatic reduction in the symptoms.

Targeting the overactive cel
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Contact: Susan Hayes
shayesconsult@earthlink.net
212-533-4472
S. Hayes Consulting
10-Oct-2002


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