GM1 GANGLIOSIDE TREATMENT OF PARKINSONS-DISEASE - AN OPEN PILOT-STUDYOF SAFETY AND EFFICACY

We performed an open-label study testing the effects of GM1 gangliosid e on 10 Parkinson's disease (PD) patients. Patients received 1,000 mg of GM1 by IV infusion after the last of three baseline functional asse ssments. Patients then self-administered GM1 at a dose of 200 mg/d, by subcutaneous injection, for 18 weeks. Under these conditions, GM1 gan glioside proved to be safe and well tolerated. There were no serious a dverse events and none of the patients developed elevated anti-GM1 ant ibody titers. Improvements on at least some functional measures were p resent in most patients, beginning after 4 to 8 weeks of GM1 treatment . When functional improvements occurred, they lasted for the duration of the study. These results suggest that GM1 ganglioside is well toler ated and may be a useful adjunct to the treatment of PD, and that a do uble-blind, placebo-controlled study is now warranted.