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GM1 Ganglioside in the Treatment of Parkinson's Diseasea



    Corresponding author
    1. Department of Pathology, Anatomy and Cell Biology, Thomas Jefferson University, 1020 Locust Street, 520 JAH, Philadelphia, Pennsylvania 19107 USA
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    Original research reported in this paper was supported by National Institutes of Health Grant AG10280, and by the F.M. Kirby Foundation, The American Parkinson's Disease Association, The National Parkinson's Foundation, Fidia Pharmaceutical Corp., and the Marion and Joseph Wesley Fund.

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ABSTRACT: Since the early 1980s, numerous studies have been reported by laboratories around the world documenting the beneficial effects of GM1 ganglioside treatment on the damaged dopamine system in various animal and in vitro models. Based on the strength of these data, the first clinical studies designed to assess the efficacy and safety of chronic GM1 use in the treatment of Parkinson's disease were performed. In a double-blind placebo-controlled study, significant improvements in GM1-treated patients were demonstrated in clinical motor ratings, timed tests of motor function, activities of daily living, and some aspects of neuropsychological functioning. Patients who have elected to continue using GM1 in an open extension trial have either continued to improve over time or have shown initial functional improvements and their disease has remained stable (i.e., no symptom progression) after two years. These results suggest that long-term use of GM1 is safe and may work to partially reverse the degenerative process in established Parkinson's disease patients.