Protecting Parkinson's brains

Parkinson's Brains

A new Emory study that uses an experimental gene therapy approach seeks to help Parkinson’s patients who continue to experience motor complications despite adequate drug therapy.

With the help of experimental gene therapy, Emory doctors are hoping to encourage brain cells under stress from Parkinson’s disease to do more nurturing.

The goal is to prod the brains of Parkinson’s disease patients to produce more of the growth factor neurturin. Neurturin is a naturally occurring protein that has been shown to protect and improve the function of damaged brain cells in animal models of Parkinson’s. Doctors think neurturin could help prevent loss of the dopamine-producing brain cells that help regulate movement and slow progression of the disease’s motor symptoms, including tremor and stiffness.

The study is aimed at Parkinson’s patients who continue to experience motor complications despite adequate drug therapy. Emory is one of 11 institutions nationwide participating in the research, which is partially funded by the Michael J. Fox Foundation for Parkinson’s Research. The clinical trial will follow 50 patients for 15 weeks up to three years after treatment to test safety and efficacy of the treatment.

In the trial, surgeons will inject a gene that encodes neurturin (clothed in a modified virus that doesn’t cause disease) directly into the substantia nigra and putamen, two key brain areas damaged in Parkinson’s. Half of the participants will undergo a placebo procedure, where nothing will be injected. If the study demonstrates the therapy to be safe and beneficial, those who receive placebo operations can receive the therapy later at no cost.

“Most available therapies for Parkinson’s disease treat symptoms and do not address the degeneration of the brain cells,” says Emory neurosurgeon and principal investigator Nicholas Boulis. “This trial takes a new approach that emphasizes the protection of brain cells and is aimed at restoring function and ultimately delaying the progression of the disease.”  

Another Emory study takes a similar approach for Alzheimer’s patients, but it uses a different gene (nerve growth factor) and targets a different part of the brain.

In previous clinical trials, surgeons have inserted growth factor directly into the brains of Parkinson’s patients with disappointing results. However, in the aftermath of those trials, they believe that the growth factors didn’t spread enough to benefit the targeted areas.

By contrast, “gene therapy would be an improvement over infusion therapy as it involves one procedure and leads to long-lasting expression,” says Emory neurologist and co-investigator Stewart Factor. —Quinn Eastman 

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