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Gene Transfer Therapy Eases Parkinson's Symptoms

Movement improved after treatment to stop death of dopamine-producing brain cells, study finds

MONDAY, April 16, 2007 (HealthDay News) -- A new kind of gene therapy treatment for Parkinson's disease shows promise, according to a new U.S. study.

The Phase I study by researchers at the University of California, San Francisco, and Rush-Presbyterian Medical Center in Chicago, in conjunction with Ceregene Inc. of San Diego, used a method called gene transfer to deliver a growth factor to the brains of 12 people with Parkinson's disease.

Previous research has found that certain growth factors can slow or stop the death of dopamine-producing brain cells. A lack of dopamine results in Parkinson's symptoms such as tremors, limb rigidity, slow movement and balance and coordination problems.

In this study, researchers used a modified virus (adeno-associated virus -- AAV) to deliver the growth factor gene neurturin into targeted brain cells. The modified virus cannot reproduce or damage brain cells. Laboratory studies have shown that neurturin helps prolong survival of dopamine-producing cells.

The researchers used multiple needle injections to deliver the AAV-neurturin through small openings in the skull and directly into the striatum, the part of the brain most deficient in dopamine. A lower dose was tested in half the patients and a higher dose in the other half.

In nine of the 12 patients for whom one-year outcome data was available, there was a 38 percent improvement in movement. There were no major adverse effects from either the low dose or high dose of AAV-neurturin. The findings warrant a larger, Phase II study, the researchers said.

The findings were expected to be presented Monday at the annual meeting of the American Association of Neurological Surgeons, in Washington, D.C.

More information

The National Parkinson Foundation has more about Parkinson's disease treatment options.

SOURCE: American Association of Neurological Surgeons, news release, April 16, 2007
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