Abstract
The present single center, double-blind, delayed start study was conducted to examine
possible symptomatic and disease-modifying effects of GM1 ganglioside in Parkinson's
disease (PD). Seventy-seven subjects with PD were randomly assigned to receive GM1
for 120 weeks (early-start group) or placebo for 24 weeks followed by GM1 for 96 weeks (delayed-start group). Washout evaluations occurred at 1 and 2 years after the end of treatment. Seventeen additional subjects who received standard-of-care
were followed for comparative information about disease progression. Primary outcome
was change from baseline Unified Parkinson's Disease Rating Scale (UPDRS) motor scores.
At week 24, the early-start group had significant improvement in UPDRS motor scores
vs. a significant worsening of scores in the delayed-start group. The early-start
group also showed a sustained benefit vs. the delayed-start group at week 72 and at
week 120. Both groups had significant symptom worsening during washout. This study
provides evidence that GM1 use for 24 weeks was superior to placebo for improving motor symptoms and that extended GM1 use
(up to 120 weeks) resulted in a lower than expected rate of symptom progression. The data from
this small study suggest that GM1 may have symptomatic and potentially disease modifying
effects on PD.
Keywords
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Article info
Publication history
Published online: November 30, 2012
Accepted:
October 24,
2012
Received in revised form:
October 8,
2012
Received:
August 29,
2012
Identification
Copyright
© 2012 Elsevier B.V. Published by Elsevier Inc. All rights reserved.
