Trial to Test Gene Therapy for Parkinson’s Disease

(National Institute on Aging, NIH)

Rush University Medical Center in Chicago, Illinois and 10 other U.S. hospitals are testing a gene therapy called CERE-120 to evaluate if it can improve the symptoms of Parkinson’s disease. CERE-120 is an experimental gene transfer drug being developed by Ceregene Inc. in San Diego, California.

The drug contains the human gene for neuturin, a naturally occurring protein also known as a neurotrophic factor. Neurosurgery is used to deliver the neuturin directly to degenerating or dying dopamine neurons in the brain. Earlier studies have shown neuturin to improve function and repair brain cells that degenerate in Parkinson’s disease.

Three previous trials demonstrated that CERE-120 was safe in 50 Parkinson’s disease patients who were followed for five years. The new Phase 2b trial will test the efficacy of CERE-120 by delivering an increased dose of the gene therapy to two areas of the brain — substantia nigra and the putamen — damaged by Parkinson’s disease. The goal of this new approach is to widen the distribution of neurturin and increase the likelihood of repairing and protecting brain cells from further degeneration due to Parkinson’s disease.

In the double-blinded trial, half of the study participants will undergo surgery to receive a dosing of CERE-120. The other half will undergo a placebo surgery. A comparison between these two groups will help distinguish the effects of CERE-120 compared to those that receive placebo. If the study results demonstrate that CERE-120 is safe and beneficial, subjects who receive placebo surgery will have the option to have a second surgery to receive a dosing of CERE-120.

Read more: Biotech Researchers Publish Parkinson’s Gene Silencing Data

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