Gene therapy study helps with Parkinson's
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A therapy developed by Neurologix Inc. shot a gene deep into the brains of people with Parkinson's disease, easing their jerking motions and tendency to freeze, according to a study sponsored by the company.
The gene acts like a factory, pumping out one of the brain chemicals lacking in people with the disorder to rebalance the substances that stimulate and inhibit activity, said Michael Kaplitt, a founder of Fort Lee, N.J.-based Neurologix and co-creator of the therapy. The study was published last week in the journal Lancet Neurology.
The therapy appeared safe and seemed to improve motor control, compared with subjects who received a placebo through a sham surgical procedure, said Jon Stoessl, a professor, head of neurology and director of the Pacific Parkinson's Research Center at the University of British Columbia in Vancouver. Dr. Stoessl, who wasn't involved in the study, said the findings should be evaluated in larger trials.
"This is the first controlled trial to show a significant improvement in the active group of a gene therapy trial," Dr. Stoessl said in a phone interview. "It appears promising and merits further investigation."
Neurologix, which has no marketed products, plans to ask the FDA this year for permission to begin an expanded trial next year that may be used to win regulatory approval, Marc Panoff, the company's chief financial officer, said in a phone interview. The company needs to raise $30 million to $40 million to conduct the study, and is talking to both investors and potential pharmaceutical industry partners to obtain funding, he said.
Walter Liskiewicz, 60, a retired oral surgeon from Jackson, Mich., who has been living with Parkinson's for 18 years, said he was in "terrible" shape before taking part in the study. "I wiggled all the time. I had trouble walking. Life sucked," he said.
After being treated in July, 2009, Mr. Liskiewicz started noticing slow, steady improvement, he said in a phone interview. Now, he walks a mile or two once or twice a week and can carry groceries into his house.
First Published March 21, 2011 12:00 am
