Can Gene Therapy Slow Progression of Parkinson's?

Phase 2 Clinical Trial at NewYork-Presbyterian/Columbia Looks at Whether Gene Therapy Approach Can Prevent Death of Dopamine Neurons in Patients With Parkinson's

Mar 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 factor–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 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.

NewYork-Presbyterian Hospital

NewYork-Presbyterian Hospital, based in New York City, is the nation's largest not-for-profit, non-sectarian hospital, with 2,353 beds. The Hospital has more than 2 million inpatient and outpatient visits in a year, including more than 220,000 visits to its emergency departments — more than any other area hospital. NewYork-Presbyterian provides state-of-the-art inpatient, ambulatory and preventive care in all areas of medicine at five major centers: NewYork-Presbyterian Hospital/Weill Cornell Medical Center, NewYork-Presbyterian Hospital/Columbia University Medical Center, NewYork-Presbyterian/Morgan Stanley Children's Hospital, NewYork-Presbyterian/The Allen Hospital and NewYork-Presbyterian Hospital/Westchester Division. One of the most comprehensive health care institutions in the world, the Hospital is committed to excellence in patient care, research, education and community service. NewYork-Presbyterian is the #1 hospital in the New York metropolitan area and is consistently ranked among the best academic medical institutions in the nation, according to U.S.News & World Report. The Hospital has academic affiliations with two of the nation's leading medical colleges: Weill Cornell Medical College and Columbia University College of Physicians and Surgeons.

Columbia University Medical Center

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