‘Novel treatment for advanced Parkinson’s disease’ found

March 17th, 2011 - 5:36 pm ICT by ANI  

Washington, March 17 (ANI): A new study has indicated that a multi-center gene therapy trial for patients with advanced Parkinson’s disease demonstrated reduced symptoms of the progressive movement disorder.

The study was designed to deliver the gene for glumatic acid decarboxylase (GAD) packaged in inert viral vectors into an area of the brain called the subthalamic nucleus. GAD makes an important inhibitory chemical called GABA. The subthalamic nucleus is abnormally activated in Parkinson’s disease and this activity leads to the debilitating movement problems. The idea of the gene therapy is that the billions of AAV-2 GAD viral vectors delivered into the subthalamic nucleus will increase GABA, thereby quieting this brain region.

A total of 45 patients were enrolled in the study. Roughly half of the patients (23) were randomized into the sham surgery arm of the study, which meant that they had a surgical procedure that did not penetrate the brain, and received infusions of saline under the skin rather than the active GAD-containing viral vectors.

Everyone was assessed at one month, three months and six months after the genes were infused. Each patient in the active treatment received about a billion viral vectors. It is not clear how long the genes will pump out GAD to make GABA.

The scientists only included patients who got bilateral infusions delivered to the correct area of the brain, the subthalamic nucleus. The final analysis included 16 patients who received active (AAV2-GAD) treatment and 21 who received the sham surgery.

“This is a completely novel treatment for advanced Parkinson’s disease,” said Andrew Feigin, lead investigator of the study, associate professor of neurology and molecular medicine at The Feinstein Institute for Medical Research in Manhasset, NY.

“The treatment was remarkably well tolerated, with mostly only mild adverse events in the AAV2-GAD treated group that were felt to be unrelated to the treatment, and completely resolved,” said Feigin.

The study has been published in Lancet Neurology. (ANI)

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