PDF is committed to using your funds efficiently to support our mission. To see how funds are spent, browse our financial information.
News In Brief
Study Suggests DBS More Effective than Meds Alone for Advanced PD
A study that compared best medical therapy to deep brain stimulation (DBS) in people with advanced PD showed that DBS may be more effective in managing Parkinson's symptoms.
Published in the August 31 edition of The New England Journal of Medicine, this study was conducted in Germany and Austria with 156 people with Parkinson's under the age of 75 who had severe motor symptoms, wearing-off episodes and dyskinesias.
Participants were randomly assigned to receive medical management or to undergo DBS in the subthalamic nucleus.
After six months, assessments showed that the DBS group had a nearly 25 percent improvement in mobility, emotional well-being and physical discomfort. The medication group remained at nearly the same level that was observed at the start of the trial.
Overall incidence of adverse effects was higher in the medication group. However, serious adverse effects, including one fatal brain hemorrhage, were more common in the DBS group.
Results Presented from Early Gene Therapy Trials
In October, two research teams announced the results from early-phase human trials of gene therapy to treat Parkinson's disease. Gene therapy is an experimental treatment method that injects a gene carried by a virus deep into the brain where Parkinson's occurs. The goal of gene therapy is to slow the course of the disease by preventing brain cells from dying.
At the Society for Neuroscience meeting, Dr. Matthew J. During of Weill Medical College of Cornell University (a PDF Center Grant recipient) presented data from a trial that tested the safety of the therapy using a gene for an enzyme related to a neurotransmitter called gamma-amino-butyric acid, or GABA, which is essential for controlling muscle movements. Dr. During and his colleagues surgically implanted one of three doses of the enzyme into the brain on one side of the subthalamic nucleus in 12 people with Parkinson's. No serious side-effects developed and by six months, patients showed improvement in their parkinsonian impairments. Brain scans showed increased GABA activity.
At the American Neurological Association meeting, Dr. William J. Marks reported on a trial led by scientists from Rush University Medical Center (another PDF Center Grant recipient) and the University of California, San Francisco. The team tested neurturin, a nerve growth factor that occurs naturally in the brain.
Researchers injected a gene carrying neurturin into the putamen of 12 people with Parkinson's. Both sides of the brain received the implant and two doses were used. No serious side-effects occurred. At six months the patients' parkinsonian signs seemed to improve.
Scientists note that while the data from both studies are encouraging, the trials are early and were focused primarily on safety, not efficacy. Also, some patients might have been experiencing the "placebo effect" often observed in surgical trials.
Risks of gene therapy include infection, bleeding and barriers to easy termination of the treatment if side-effects occur. Follow up on the participants will continue, and controlled Phase II trials to assess efficacy are in the planning stages.