GM-1 Ganglioside

Definition

The drug GM-1 ganglioside is an experimental drug for acute injury, including stroke and spinal injury.

Description

Acute spinal cord injury is an unexpected, catastrophic event, the consequences of which often persist for the life of the patient. The incidence of spinal cord injury in the U.S. is over 10,000 cases per year.

A study in 1991 reported in the New England Journal of Medicine reported the results of a prospective, randomized, placebo-controlled, double-blind drug trial of GM-1 ganglioside in patients with spinal cord injuries, with a follow-up period of one year after the injury to assess recovery. The ganglioside used in the study was Sygen supplied by Fidia Laboratories.

The study was claimed by the authors as representing a dramatic increase in neurologic function, with the majority of the patients changing from paralyzed to ambulatory status.

The marked improvement in the recovery of paralyzed motor groups, along with the similar recovery of the paretic muscles in the two treatment groups, indicated that the observed effect of GM-1 occurs by conversion of motor groups initially paralyzed into those with useful motor functions after one year. The study concluded that GM-1 is safe to administer in spinal cord injury and enhances the recovery of neurologic function after one year, but a larger study should be conducted to confirm its clinical benefit and safety.

In response to the publication of the research study, a letter in the New England Journal of Medicine argued against the efficacy of GM-1 (Sygen) of the Fidia Pharmaceutical Corporation and noted that the study failed to show any improvement in motor function after spinal cord injury.

Current Research

Spinal cord injury

Spinal cord injury affords limited natural recovery and only a few generally ineffective treatment options. Recent publications have reported enhanced neurologic recovery with the use of methylprednisolone and GM-1 ganglioside. The results of the Maryland GM-1 Ganglioside Study reported a significant drug effect. That study formed the basis for the larger placebo-controlled multicentered study using Sygen GM-1 in patients with acute spinal cord injury.

Cerebral Ischemia

The concept of neuroprotection relies on the principle that delayed neuronal injury occurs after ischemia (loss of blood flow and oxygenation). In animal models of cerebral ischemia, numerous preclinical studies have demonstrated various drugs to be neuroprotective.

A number of drugs have been studied in humans. At least ten classes of neuroprotective agents have reached phase III efficacy trials but have shown mixed results. They include GM-1 ganglioside, calcium channel antagonists, NMDA receptor antagonists, lubeluzole, CDP-choline, anti-intercellular adhesion molecule-1 (ICAM-1), and the sodium channel antagonist fosphenytoin. In the future, clinicians may have a choice of treatments for acute ischemic stroke.

The efficacy and safety of GM-1 ganglioside is still under study.

Questions to Ask Your Doctor

Has GM-1 Ganglioside been approved by the FDA?

What are the risks?

What is the cost?