Potential New Drug for Parkinson’s Disease Tested at Jefferson

A clinical trial involving an experimental medication for Parkinson’s disease showed positive results in a group of 77 patients.

The drug GM1 ganglioside, a substance naturally enriched in the brain, appeared to act as a “neuroprotective” and a “neurorestorative” agent to improve symptoms and, over an extended period of time, slow the progression of symptoms.

The researchers enrolled the subjects and followed them over a 120-week period. In addition they followed 17 subjects who received current standard of care treatment for comparison.

“Our data suggest that GM1 ganglioside has the potential to have symptomatic and disease-modifying effects on Parkinson’s disease,” said Jay S. Schneider, PhD, Director of the Parkinson’s Disease Research Unit and Professor in the Department of Pathology, Anatomy and Cell Biology and the Department of Neurology at Jefferson.

“If this is substantiated in a larger clinical study, GM1 could provide significant benefit for Parkinson’s disease patients,” Dr. Schneider added.

That is particularly true, he said, because none of the currently available medications for the movement disorder “has been shown unequivocally to slow disease progression.”

Parkinson’s disease affects over 1 million people in the U.S. and is diagnosed in some 60,000 adults every year. The symptoms include tremors, slowness in movement, difficulty initiating movements, difficulty walking, balance problems and decrease in speech volume and facial expression.

GM1 ganglioside is a chemical that is normally found in the brain and part of the outer covering of nerve cells. It plays important roles in neuron development and survival and controls a wide variety of cell activities. In preclinical studies GM1 was found to rescue damaged neurons and increase dopamine levels.

GM1 effects many different cellular functions, so trying it in Parkinson’s disease, rather than focusing on a specific potential disease mechanism was logical, said Dr. Schneider.

“Instead of a magic bullet, we think of it like a magic shotgun,” he said. “This study was truly a success of translational research.”

The Movement Disorders Program

Jefferson’s Parkinson’s Disease and Movement Disorders Program serves as a tertiary care center for the Philadelphia region’s most complex and challenging cases of chronic neurological disease. The Program offers patient-centered care incorporating the latest medical, surgical, psychological and rehabilitative therapies.

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