Gene Therapy for Parkinson's?

By Kate McHugh, Ivanhoe Health Correspondent

ORLANDO (Ivanhoe Newswire) -- What began as a study on the effectiveness of gene therapy on Parkinson's patients may have more far-reaching impacts for sufferers of many neurological diseases.

Researchers from the New York-Presbyterian Hospital/Weill Cornell Medical Center tested the effectiveness of injecting a harmless gene-bearing virus into the brains of 12 patients with advanced Parkinson's disease. According to lead researcher Michael Kaplitt, M.D., Ph.D., the results were promising.

Three months after the gene therapy injections, Dr. Kaplitt says the Parkinson's patients showed between 25-percent and 30-percent improvement in motor function when they were off their regular Parkinson's medications and between 40-percent and 65-percent improvement when they were on their medications. The improvements were maintained throughout the 12 month study. Results such as these, Dr. Kaplitt says, show the potential to slow the progression of Parkinson's in the brain but also in neurological diseases across the board.

"Our hope is that…we will not only see this therapy develop and evolve, but we will also see a whole host of other therapies for other diseases," Dr. Kaplitt told Ivanhoe. Similar trials on epilepsy and Alzheimer's are beginning this fall, according to Dr. Kaplitt. Depression and even obesity are among the diseases he says could benefit from gene therapy.

Dr. Kaplitt says this is the first time gene therapy was tested on an adult human brain. Not only did the treatment prove to be safe, but researchers were able to chart significant change in the genetic make-up of the patient's own brain cells. Previous trials on animals, including primates, man's closest animal "relative," suggest the injected gene stays active for years.

SOURCE: Ivanhoe interview with Michael Kaplitt, M.D.; The Lancet, published online June 23, 2007