New York, NY - March 24, 2011 - An experimental therapy that may prevent the death of dopamine neurons in patients with Parkinson's disease is now being tested at NewYork-Presbyterian Hospital/Columbia University Medical Center and 10 other leading medical centers around the country.
"CERE-120 therapy is the only neurotrophic factorâ€“based gene therapy currently in clinical trials that has the potential to slow down the progression of Parkinson's," says Dr. Blair Ford, professor of clinical neurology, neurologist and the principal investigator of the study at NewYork-Presbyterian Hospital/Columbia University Medical Center. "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 cells by delivering the gene for neurturin, a naturally occurring protein also known as a neurotrophic factor, directly to the brain. In laboratory experiments, neurturin was shown to repair damaged nerve cells, restore their function and help keep them alive.
Results from a previous study published in the journal Lancet Neurology (Marks et al., 2010) suggest small improvements in Parkinson's symptoms when CERE-120 is injected into only one part of the brain. In the current Phase 2 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 randomized 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, including ways to enroll, is available in the clinicaltrials.gov study registry.
The CERE-120 gene therapy study is the latest in a long history of Parkinson's disease clinical trials at NewYork Presbyterian/Columbia — 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.