The Therapeutic Role of Gangliosides in Neurological Disorders.

Numerous in vitro and in vivo experimental animal studies have demonstrated that gangliosides, particularly GMI ganglioside (siagoside), may stimulate or accelerate the repair of peripheral and central nervous system neurons after various types of damage. Clinical studies of GMl in peripheral neuropathies and stroke, disorders in which the effects of GM 1 have been studied most extensively, have yielded inconsistent results. Problems of inadequate study design, inclusion of heterogenous clinical popUlations and variations in dosage, duration of treatment and timing of initial administration make it difficult to compare results from individual studies and to conclusively assess efficacy of GMl treatment. Therefore, further clinical studies of gangliosides seem warranted.Despite efforts to link gangliosides with the development of Guillain-Barre syndrome, there are no valid data to support such an association. Gangliosides, in particular GM I, are well tolerated after repeated administration to humans and do not appear to be immunogenic.GM1 may be a useful therapeutic agent if administered to particular patients with a specific spectrum of symptoms and disease severity. In addition, to be effective the agents need to be administered in appropriate dosages and at an appropriate time after neuronal injury. Human clinical trials of gangliosides should continue cautiously and with restraint.