Is ganglioside GM1 effective in the treatment of stroke?

Nine double-blind, placebo-controlled clinical trials involving 784 patients with nonhaemorrhagic stroke have been reviewed to investigate the clinical efficacy of ganglioside GM1 in this disorder. The evaluated data show a statistically significant, but transient, acceleration in recovery from neurological deficits in 63% of all GM1-treated patients during the first 2 weeks after the event compared with placebo. Both studies in which the first administration of GM1 occurred within 48 hours after stroke demonstrated a statistically significant benefit in neurological recovery for the GM1-treated patients, suggesting an advantage of early administration. However, quantitative neurological assessments of GM1- and placebo-treated patients between 2 weeks and 6 months, disability scores and mortality rates failed to show significant differences. The problems and limitations of the trials (differences in patient populations studied, lack of standardised assessment criteria, etc.) are discussed, and potential mechanisms of GM1 action and factors that influence treatment results (e.g. early administration) are evaluated.