First in Vivo Gene Therapy in Adult Brain for Parkinson's

As part of a Phase I clinical trial, surgeons recently performed the first gene therapy for Parkinson's disease on a 55-year-old man. The patient was expected to return home just two days after the surgery.

In the procedure, surgeons pinpointed the optimal location in the patient's brain using information from an advanced 3T magnetic resonance (MR) image, which was merged with a computed tomography (CT) scan. The final target was confirmed by using fine electrical probes that identified the signature pattern of electrical activity of individual cells within the brain. During this process, the patient was awake and not medicated, since medication and anesthesia can confuse the electrical information. Once the target was confirmed, an adeno-associated virus (AAV) was slowly delivered through a very fine catheter.

After a 90-minute infusion, the catheter was removed, the skin was closed, and the patient was sent to the recovery room. The AAV is the means by which the glutamic acid decarboxylase (GAD) gene enters the appropriate brain cells and begins production of a protein that produces GABA, a molecule that is released by nerve cells to inhibit or dampen activity.

The five-hour surgery was performed by Dr. Michael G. Kaplitt, assistant professor of nuerologic surgery at Weill Cornell Medical College (New York, NY, USA; www.med.cornell.edu). "The goal of our gene therapy approach is to ‘reset' a specific group of cells that have become overactive in an affected part of the brain, causing the impaired movements associated with Parkinson's disease,” said Dr. Kaplitt.




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Weill Cornell Med. College