1st Gene Therapy Trial Effective Against Parkinson's

Small study showed symptoms were eased, but treatment needs larger testing, researchers say.

By Amanda Gardner
HealthDay Reporter
Thursday, June 21, 2007; 12:00 AM

Copyright © 2007 ScoutNews, LLC. All rights reserved.

THURSDAY, June 21 (HealthDay News) -- In an early trial, gene therapy has proven both safe and effective in easing the symptoms of Parkinson's disease, researchers say.

A dozen patients experienced a 25 percent to 30 percent improvement in their total symptoms without any side effects, the U.S. team noted in the June 23 issue of The Lancet.

"Safety and tolerability were met with flying colors. On that level, we are extremely encouraged," said Dr. Matthew During, senior author of the study and professor of molecular biology, immunology and medical genetics at Ohio State University. "All in all, we're very excited, but we have to be a little bit cautious, and we need to do a definitive study with the proper controls."

During completed the study while on the faculty of Weill Medical College of Cornell University in New York City. The trial was conducted with researchers from the Feinstein Institute for Medical Research, part of the North Shore-LIJ Health System, in New York. The study was funded by Neurologix, which was co-founded by During and another study author, Dr. Michael Kaplitt. The two remain as consultants to the company, and Kaplitt's father is chairman of the board of Neurologix.

Parkinson's disease affects some 1.5 million Americans, most of them over the age of 65, although younger patients are also affected, including actor and Parkinson's research advocate Michael J. Fox.

The illness is characterized by a loss of dopamine-producing brain cells, resulting in problems with motor function such as tremors, limb rigidity, slow movement and balance and coordination problems.

There are some treatments but no cure, and, as During pointe out, current treatments are far from ideal.

Patients eventually become resistant to drugs. Deep brain stimulation, which involves placing an electrode deep into the brain, has helped some patients who no longer respond to drugs. But the therapy has side effects and is only partially effective.

"There are wires and a huge battery pack in the chest. It's a bit Frankensteinian, and patients hate it," During said. "There's about a 30 to 40 percent adverse event rate. Although it works, it comes at a significant cost."