RESEARCH & PUBLICATIONS
New Gene Therapy Trial for Parkinson's Disease Now Underway
An experimental therapy that may prevent the death of dopamine neurons in Parkinson's disease is now being tested at Columbia University Medical Center and 10 other leading medical centers around the country.
"CERE-120 therapy is the only gene therapy currently in clinical trials that has the potential to slow down the progression of Parkinson's," says Blair Ford, MD, Professor of Clinical Neurology and the principal investigator of the study at Columbia. Other gene therapies currently under investigation aim to reduce symptoms, but are not designed to help prevent the death of nerve cells in the brain."
CERE-120 attempts to protect nerve cell by delivering neurturin, a naturally occurring protein also known as a neurotrophic factor, directly to the brain. Neurotrophic factors help keep neurons healthy, and in laboratory experiments, neurturin in particular was shown to repair the dopamine neurons damaged by Parkinson's, restore their function and help keep them alive.
The clinical study is partially funded by a LEAPS grant from the Michael J. Fox Foundation and is sponsored by Ceregene, the biopharmaceutical company that developed CERE-120. More information about the study is available in the clinicaltrails.gov study registry.
A previous study of CERE-120, published in the journal Lancet Neurology (Marks et al., 2010) has found small improvements in Parkinson's symptoms when CERE-120 is injected into only one part of the brain. In the current study, neurosurgeons will instead inject CERE-120 into two regions of the brain most affected by Parkinson's, the substantia nigra and the putamen. Once in the brain, the neurturin gene should begin producing a lifelong supply of neurturin. This new approach intends to expose both ends of the dopamine neurons to neurturin and will hopefully lead to more meaningful improvement in symptoms.
Dr. Ford cautions that CERE-120 is still experimental and comes with risks, including those associated with neurosurgery. Viral vectors similar to that used in CERE-120 have been used in many other experimental gene therapies and have not caused serious side effects or disease, but there still may be unknown risks.
The CERE-120 gene therapy study is the latest in a long history of Parkinson's disease clinical trials at Columbia/New York Presbyterian Hospital – from the earliest trials of l-dopa to the trials of deep brain stimulation, embryonic cell implants, and virtually every compound used in the treatment of Parkinson's disease – that makes the institution an internationally recognized leader in patient care and research.
- Ford B, Winfield L, Frucht PE, Cheringal J, Du E, Pullman S, Yu Q, Fahn S, Cote LJ, McKhann GM, Goodman RG. Subthalamic nucleus stimulation in Parkinson's disease: blinded evaluations at one year follow-up (submitted).
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- Surgery for Parkinson’s Disease: A guide for patients, families and caregivers. Parkinson’s Disease Foundation, 2003.
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- Ford B, Greene P, Louis ED, Petzinger G, Bressman SB, Goodman R, Brin MF, Sadiq S, Fahn S. Use of intrathecal baclofen in dystonia. Arch Neurol 1996;53:1241-1246.
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September 25, 2012