GM1 gangliosides in the treatment of spinal cord injury: report of preliminary data analysis.

A prospective, randomized, placebo-controlled, double-blinded spinal cord injury GM1 ganglioside drug trial was completed. Of the 37 patients entered over a 16 month period, 34 patients (23 cervical and 11 thoracic injuries) received the test drug protocol and completed the one year follow up period. The neurologic recovery was quantified by serial measurement of the ASIA motor score throughout the acute hospital course and one year long follow up period. The primary variable used to assess neurologic recovery was the difference in the ASIA motor score from the admission value to the value at one year. The GM1 group had an average motor recovery of 36.9 points whereas the placebo group had an average change of 21.6 points (t-test difference, p = 0.088). Analysis of the secondary variable, the area under the ASIA motor score versus the logarithm of time, and the use of rank order nonparametric statistic on both the primary and secondary variables to sort neurologic recovery obtained similar statistical differences between the GM1 and placebo treatment groups. Randomization imbalances in baseline severity of injury and division of cervical and thoracic injury occurred in the trial. Because of this fact and the small sample size of the study verification of these results by a larger study is required.