Public Release: 

Jefferson scientist begins clinical trial to study promising Parkinson's disease drug

Thomas Jefferson University

GM1 ganglioside may improve symptoms, delay disease advance

Scientists at Jefferson Medical College, 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 in some cases actually restore damaged brain cells in Parkinson's disease patients.

Jay S. Schneider, Ph.D., professor of pathology, cell biology and anatomy and neurology at Thomas Jefferson University in Philadelphia, 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.

"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 SygenR, 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 in Philadelphia 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. The participants, patients with mild to moderate Parkinson's disease, will be divided into three groups of 50 each. One group will receive the drug during the first two study phases. Another 50 will receive a placebo during phase one, and will be allowed to take the drug later in the second trial phase. A third control group of 50 will receive neither the study medication nor placebo, but will receive standard Parkinson's therapy. This latter group will have the option of receiving study medication when the study period ends.

"We're hoping that in the first six months of the study we'll see the same kind of symptomatic improvement we've seen in previous studies with GM1, as well as a small increase in the number of dopamine terminals shown in subsequent SPECT scans," Dr. Schneider says.

"The second part of the trial will examine whether long-term use of GM1 actually influences the course of Parkinson's," he says. This study phase will address the question of whether a difference exists between the rate of symptom advancement and loss of dopamine terminals in GM1 versus non-GM1-treated patients.


More than one million people in the United States suffer from Parkinson's disease. It commonly strikes people over 50. Symptoms include hand tremor, slowness in movement, difficulty initiating movements, difficulty walking, shuffling feet, decrease in speech volume, fatigue, and balance problems.

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-NOW.

Steve Benowitz or Phyllis Fisher
After Hours: 215-955-6060

Lynda Woodworth
Fidia/Dragonette, Inc.

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