Past and current clinical studies with GM-1 ganglioside in acute spinal cord injury.

Abstract

Two recent prospective, randomized, placebo-controlled, double-blinded clinical drug studies in acute spinal cord injury have reported enhancement of neurologic recovery of motor function. The drugs investigated in these studies were methylprednisolone and GM-1 ganglioside. Before these studies, the treatment of patients with spinal cord injuries had been restricted to prevention of further injury to the spinal cord, limiting secondary damage following the initial injury, increasing the patient's ability to function through intensive rehabilitation, and facilitating any spontaneous recovery of neurologic function. Methylprednisolone is a steroid administered at very high levels, and GM-1 is a complex acidic glycolipid found at high levels in cell membranes in the mammalian central nervous system with known neuroprotective and neurofunctional restoration potential. This article summarizes the previously reported Maryland GM-1 clinical trial and presents the clinical and statistical design of a larger clinical trial being conducted with the purpose of verifying a beneficial GM-1 drug effect when administered with methylprednisolone in acute spinal cord injury.