Gene Therapy May Mean Hope for Parkinson’s Patients

By Lindsay Braun, Ivanhoe Health Correspondent

ORLANDO, Fla. (Ivanhoe Newswire) -- Brain scans confirm that an experimental gene therapy treatment is effective in normalizing brain function in Parkinson’s patients.

Researchers from The Feinstein Institute for Medical Research and Weill Cornell Medical Center collaborated to study the outcome of introducing genes into the subthalmic nucleus of the brain.

“The genes were delivered using a viral vector that was infused through a craniotomy, which means they were surgically implanted in the brain,” Andrew Feigin, M.D., assistant professor of medicine and NYU School of Medicine and The Feinstein Institute for Medical Research told Ivanhoe.

The twelve patients who participated in the study received the gene therapy to one side of their brain to reduce risk and to make for an easier assessment. The side of the brain to receive treatment was decided based on which side of the patients body was most affected by the disease.

The therapy was designed to regulate the patient’s motor network in the brain, which controls movement. Position emission tomography (PET) scans six months after the therapy confirmed the therapy did impact the motor network, and made no change in the cognitive network.

“We were pleased with how well tolerated the procedure was, and that the effects of the procedure could still be seen on the PET scans 1 year after surgery. I wouldn’t say we were surprised by these things, but they were unknowns before we did this study,” Dr. Feigin said.

Although the results of this study are exciting, researchers say there is still a lot of work to be done. “I think that it is important to emphasize that this was an initial study that was primarily aimed at assessing safety issues. Future randomized blinded studies will be needed to confirm our efficacy findings, “ Dr. Feigin said.

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