PARKINSONS-DISEASE - IMPROVED FUNCTION WITH GM1 GANGLIOSIDE TREATMENTIN A RANDOMIZED PLACEBO-CONTROLLED STUDY

Background/Objective: Studies in animal models of Parkinson's disease (PD) suggest that GM1 ganglioside treatment can restore neurologic and dopaminergic function. In view of positive preclinical findings and t he results of a previous open-label study demonstrating efficacy of GM 1 in PD patients, this study compared effects of GM1 ganglioside and p lacebo on motor functions in PD patients. Methods: Forty-five patients with mild to moderate PD were studied. The primary efficacy measure w as change in the Unified Parkinson's Disease Rating Scale (UPDRS) moto r score. After three independent baseline assessments, patients receiv ed IV infusion of the test drug (1,000 mg GM1 or placebo) and then sel f-administered either GM1 or placebo twice daily (200 mg/day, subcutan eously) for 16 weeks. Patients were examined during monthly follow-up visits. Results: There was a significant difference between groups in UPDRS motor scores at 16 weeks (p = 0.0001). The activities of daily l iving portion of the UPDRS (off-period assessment) also showed a signi ficant effect in favor of the GM1-treated patients (p = 0.04). GM1-tre ated patients also had significantly greater mean improvements than pl acebo-treated patients in performance of timed motor tests including t ests of arm, hand, and foot movements, and walking. GM1 was well toler ated and no serious adverse events were reported. Conclusions: This st udy demonstrates that GM1 ganglioside treatment enhances neurologic fu nction significantly in PD patients. Further study is warranted to eva luate long-term effects of GM1 in PD patients and to elucidate further the mechanisms underlying patient improvements.