Scientists at Jefferson Medical College (JMC), armed with a newly awarded $2.4 million grant from the National Institutes of Health, hope to find out whether a promising drug, GM1 ganglioside, can improve symptoms, delay disease progression, and actually restore damaged brain cells in some patients with Parkinson’s disease.

Jay S. Schneider, PhD, Professor of Pathology, Cell Biology and Anatomy and Neurology at JMC, will lead a five-year clinical trial involving 150 patients. The study will compare the effectiveness of GM1 ganglioside – a naturally occurring substance in the nerve cell’s membrane that plays an important role in cell growth, development, and repair – to standard Parkinson’s disease treatments, which improve symptoms but do not alter the disease process.

Therapy under study may alter disease progression
“We hope that we will be able to do something no one else has done before – to stimulate remaining brain cells to sprout new nerve endings and rescue other cells that might otherwise die,” says Dr. Schneider, who last year published evidence in the journal Neurology showing that GM1 may improve symptoms in Parkinson’s disease patients and perhaps even help slow progression of the disease.

“If we are able to show that we’ve stimulated repair and regrowth in humans as we and others have done in the laboratory, we will have evidence for the first time of a therapy that can help restore the part of the nervous system damaged by a neurodegenerative disease,” Dr. Schneider says.

Fidia SpA of Abano Terme, Italy, which manufactures GM1 ganglioside under the name SygenŽ, will supply the drug for the study.

“Current therapies for Parkinson’s disease treat only the symptoms and do little to address the underlying disease process,” Dr. Schneider explains. “Despite the fact that you can alleviate some symptoms for a time, the disease process continues. It’s a progressive neurodegenerative disorder. GM1 has the potential to be a disease-altering therapy.”

The Jefferson team is collaborating with scientists at the University of Pennsylvania to perform a special type of imaging called single photon emission computed tomography, or SPECT. SPECT will allow the team to visualize the number of dopamine terminals in the striatum, the part of the brain that receives dopamine from the substantia nigra, which is the brain region that dies in Parkinson’s disease. Comparing patient symptoms and the number of dopamine terminals is a significant advance, notes Dr. Schneider. “This technology will give us important new insights into the relationship between the expression of the symptoms of Parkinson’s disease and the actual amount of dopamine terminals in the brain and will help us better track the progression of the disease.”

The study is a randomized, double-blind placebo controlled trial in which neither the researchers nor the patients know who receives the drug.

Fidia SpA is an Italian research-based company with an American subsidiary, Fidia Pharmaceutical Corporation, in Washington, D.C.

For more information about participating in the clinical trial, please call 1-800-JEFF-NOWhttp://www.jeffersonhealth.org/jeffnow/jeffnow.html.

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