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.
