Gene therapy for Parkinson's deemed safe


Nation Digest

September 28, 2005|By NEWSDAY

NEW YORK -- A novel gene therapy technique is safe and effective at staving off worsening symptoms of Parkinson's disease, according to the first scientific review of a dozen patients who have received the treatment over the last two years.

The patients are in advanced stages of the illness and were no longer responding to medicines when they signed on for the experimental therapy.

On Monday, one of the study investigators, Dr. Andrew Feigin of North Shore University Hospital in Manhasset, N.Y., told colleagues at a meeting on movement disorders in San Diego that there have been no problems with the technique, and that patients had a 27 percent improvement in symptoms.

Brain scans also revealed that the treatment was working. A normal brain scan shows overactivity in areas hit hard by the disease. The patients' scans showed a quieting of these areas, on the side of the brain where the genes were infused.

The novel strategy included packing genes that make an inhibitory chemical called GABA into noninfectious viruses and delivering it to a specific brain region that regulates dopamine. Parkinson's patients have depleted stores of dopamine, a chemical that regulates movement. The idea behind gene therapy is to bypass the dying dopamine-containing neurons, and use the GABA-containing cells.

"This seems to control the flow of information to the rest of the brain," said Dr. Michael Kaplitt, the study's lead surgeon.

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