News Release: Research , School of Medicine

Mar. 23,  2011

Researchers Using Gene Therapy to Treat Parkinson's Disease

News Article ImageNicholas Boulis, MD, associate professor of neurosurgery at Emory School of Medicine and principal investigator of the CERE-120 study at Emory.

ATLANTA - Researchers at Emory University are participating in a clinical trial to test the benefits and effectiveness of gene therapy to treat patients with Parkinson’s disease.

The Phase II study is evaluating whether a neurotrophic factor called neurturin improves the symptoms of Parkinson’s disease (PD). Neurturin is a human gene and naturally occurring protein that has been shown to protect and improve the function of damaged brain cells in animal models of PD.  

Parkinson’s disease is a neurodegenerative disease characterized by the loss of the dopamine-producing brain cells that help regulate movement. PD patients are unable to control body movements and often have body tremors or trembling of the hands, arms and legs and difficulty with walking, movement and coordination.    

This study uses an experimental gene transfer drug, CERE-120. It is injected into the area of the brain that produces dopamine and where cells are not functioning well or are dying from PD, causing these cells to make neurturin hence protecting themselves from the disease.  CERE-120 is also delivered to the part of the brain that uses the dopamine in order to maintain the health of connections between this structure and the dopamine-producing cells.  

Previous studies show that CERE-120 is safe in animals and in more than 50 Parkinson’s disease patients followed for up to five years. The goal of this new study is to assure wider distribution of neurturin, delivering CERE-120 directly to the substantia nigra and putamen, the two key brain areas damaged in PD and ultimately increasing the likelihood of repairing and protecting the degenerating cells.  

“Most available therapies for Parkinson’s disease treat symptoms and do not address the degeneration of the brain cells,” says Nicholas Boulis, MD, associate professor of neurosurgery at Emory School of Medicine and principal investigator of the CERE-120 study at Emory. “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.”   

Emory is one of eleven institutions nationwide and the only one in Georgia participating in this trial - which seeks to enroll approximately 50 people with idiopathic Parkinson's disease.  

Half of the subjects will undergo surgery to receive CERE-120 and the other half will undergo a "placebo surgery” where no medication will be injected. Participants of the randomized study, partially funded by Ceregene, Inc. and the Michael J. Fox Foundation for Parkinson’s Research, will be followed for three years after surgery.    

If the study results demonstrate that CERE-120 is safe and beneficial, subjects who receive placebo surgery will receive CERE-120 at no cost, as long as they remain medically fit to undergo surgery.   

For more information about the study, please contact Emory HealthConnectionSM at 404-778-7777 or 1-800-75-EMORY.  Additional study information can also be found at http://www.clinicaltrials.gov/ct2/show/NCT00985517.

 

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The Robert W. Woodruff Health Sciences Center of Emory University is an academic health science and service center focused on missions of teaching, research, health care and public service.

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