Promising drug slows down progression of Parkinson's disease
Parkinson's patients treated with the experimental drug GM1 ganglioside showed improved symptoms during a two and a half-year trial, according to Thomas Jefferson University researchers.
Although the precise mechanisms of action of this drug are still unclear, the drug may protect patients' dopamine-producing neurons from dying and at least partially restore their function, thereby increasing levels of dopamine, the key neurochemical missing in the brain of Parkinson's patients.
The research team, led by senior author Jay S. Schneider, Ph.D., 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, found that administration of GM1 ganglioside, a substance naturally enriched in the brain that may be diminished in Parkinson's disease brains, acted as a "neuroprotective" and a "neurorestorative" agent to improve symptoms and over an extended period of time slow the progression of symptoms.
What's more, once the study participants went off the drug, their disease worsened. The study enrolled 77 subjects and followed them over a 120-week period and also followed 17 subjects who received current standard of care treatment for comparison.
"The drugs currently available for Parkinson's disease are designed to treat symptoms and to improve function, but at this time there is no drug that has been shown unequivocally to slow disease progression," said Dr. Schneider.
"Our data suggest that GM1 ganglioside has the potential to have symptomatic and disease-modifying effects on Parkinson's disease. If this is substantiated in a larger clinical study, GM1 could provide significant benefit for Parkinson's disease patients."
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 modulates a wide variety of cell activities. GM1 has been found to rescue damaged neurons and increase dopamine levels in pre-clinical studies, and has been suggested to have beneficial effects in other neurodegenerative conditions.
Because GM1 has effects on many different cellular functions, it seemed a logical approach to try using a drug like GM1 ganglioside to modify the pathological processes occurring in Parkinson's disease, rather than focusing on a specific potential disease mechanism, 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 study has been published online in the Journal of the Neurological Sciences.