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Parkinson's disease symptoms improve with gene transfer drug CERE-120

Kathleen Blanchard's picture
Gene therapy for Parkinson's disease

An experimental gene transfer drug called CERE-120 is being tested by scientists for treating symptoms of Parkinson's disease. The drug was developed by Ceregene, Inc and contains a naturally occurring protein called neuturin that can restore and protect brain cells damaged by the disease and is being tested by researchers at Rush University Medical Center, on of 11 sites in the U.S. involved in the clinical trials.

Parkinson's disease gene transfer drug safe, effective in trials

The gene therapy drug is given directly into dopamine neurons that are destroyed by Parkinson's disease, leading to tremors, stiffening of the limbs and body, slow movements and loss of balance. i

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According to Dr. Christopher Goetz, director of the Parkinson’s Disease and Movement Disorders program at Rush University Medical Center and site principal investigator of the study, “The preliminary data in the Phase I study are encouraging, and the first Phase II trial showed improvement in patients who were assessed under blinded conditions at 15-18 months post treatment."

The new Phase IIb trial will compare the effect of CERE-120 given in higher doses. The researchers will study the effect of the drug on the substantia nigra and the putamen brain areas that are also damaged by Parkinson's disease in patients receiving surgery and drug delivery and a placebo group.

Patients given placebo will have the option of receiving the gene therapy if the trial is successful. Previous studies included 50 patients with Parkinson's disease, followed for five years. Dr. Goetz explains the urgent need for new therapies that can help patients with Parkinson's disease regain function but also prevent the disease from progressing. For more information about the clinical trial, and for contact information, visit the Rush University website.