Promising Developments in Neurotrophic Gene Therapy for Parkinson’s Disease

Promising Developments in Neurotrophic Gene Therapy for Parkinson’s Disease
Raymond Bartus, Ph.D. and Joao Siffert, M.D.
NWPF.org - Ceregene, Inc., a biopharmaceutical company, is progressing with enrollment and dosing in its CERE-120 (AAV2-neurturin) Phase 2b multi-center, placebo (sham)-surgery, double-blinded controlled trial in Parkinson's disease.

The purpose of this Phase 2b study is to evaluate the safety and potential benefits of CERE-120 (AAV2-neurturin) in the treatment of Parkinson's disease. CERE-120 is an experimental biological drug that uses gene transfer technology to deliver human neurturin, a neurotrophic (growth) factor, directly into the brain areas most affected by Parkinson’s disease – the dopamine producing neurons. Similar to other growth factors (such as GDNF), neurturin has been shown in laboratory tests to restore function and protect brain cells from further damage.

CERE-120 has been carefully studied in laboratory animals without any toxicity, even following very high doses. In addition, in animals with neuronal degeneration and motor deficits similar to Parkinson's disease, CERE-120 was shown to protect brain cells and restore their function.

Three clinical studies were then successfully conducted in people with Parkinson’s disease, collectively providing evidence that CERE-120 can generally be delivered safely and may possibly reduce Parkinson’s symptoms (Marks et al., Lancet Neurol, 2008; Marks et al., Lancet Neurol 2010; Siffert et al. Neurology, 2011 (abstract))

This current trial has incorporated knowledge gained from the three previous trials and is intended to more clearly establish benefit of CERE-120, while further supporting its safety (Bartus et al., Mov Disord 2011). This study is actively enrolling participants in 11 centers across the United States. These sites are among the best movement disorders centers in the country and include Baylor College of Medicine (Houston, TX), Beth Israel Medical Center (New York, NY), Columbia University Medical Center (New York, NY), Duke University School of Medicine (Durham, NC), Emory University Hospital (Atlanta, GA), Mount Sinai Medical Center (New York, NY), Rush University Medical Center (Chicago, IL), Stanford University School of Medicine (Palo Alto, CA), University of Alabama (Birmingham, AL), University of California (San Francisco, CA) and University of Pennsylvania Hospital (Philadelphia, PA).

Study participants are randomly assigned (like the flip of a coin) to either receive CERE-120 or undergo a “placebo” (sham) surgery (where minimal surgical intervention occurs and no medication is injected). After the study procedure, participants are followed for approximately 18 months in a double blind fashion and up to 3 years for safety tests. To date, approximately 50% of the 52 subjects have enrolled and many others are in the process of being enrolled. It is anticipated that enrollment will be completed in the fall of 2011.

Once the Phase 2 study is completed (anticipated in early 2013), and if there is a benefit of CERE-120, participants who had a placebo surgery will be offered CERE-120 at no cost and prior to commercial availability of CERE-120.

For more information about the study, log on towww.ceregene.com or call toll free at 1-855-CERE120 (1-855-237-3120)