Gangliosides are sialic acid-containing glycosphingolipids that are naturally present in the plasma membrane of most vertebrate cells, particularly in the nervous system where they constitute up to 10% of the total lipid mass in the brain.
Gangliosides play important roles in many physiological processes including cell differentiation, apoptosis, neuronal protection and neutrotrophins release.1 TRB Chemedica has dedicated significant research resources over many years to better understand the mechanism of action of GM1 (monosialotetrahexosylganglioside) in neurons and astrocytes to evaluate its potential in the treatment of neurological disorders.
Since the 1970s, the neurotrophic and neuroprotective properties of GM1 have been supported and demonstrated by cell culture and animal studies, demonstrating the potential role of GM1 to slow or even reverse the progression of a wide range of neurodegenerative conditions.2
Data generated by these investigations inspired clinicians in the USA and other countries to conduct clinical trials in patients with central nervous system damage due to stroke3, spinal cord injury4, Alzheimer’s disease5 and Parkinson’s disease6. Promising results suggest that the long-term administration of GM1 ganglioside in these patients is safe and provides clinical benefits.
- Fiumelli H, Allaman I, Martin J-L, Magistretti P. Effects of GM1 on activation of signaling pathways in neurons and astrocytes, dependence of astrocyte-neuron interactions. Neuroscience; San Diego, CA2016.
- Aureli M, Mauri L, Ciampa MG, Prinetti A, Toffano G, et al. GM1 Ganglioside: Past Studies and Future Potential. Mol Neurobiol. 2016;53(3):1824-1842.
- Alter M, and the SASS investigators. Ganglioside GM1 in acute ischemic stroke. The SASS trial. Stroke. 1994;25(6):1141-8.
- Geisler FH, Dorsey FC, Coleman WP. Recovery of motor function after spinal cord injury – A randomized placebo – controlled trial with GM1 ganglioside. N Engl J Med. 1991;324(26):1829-38.
- Svennerholm L, Brane G, Karlsson I, Lekman A, Ramström I, Wikkelsö C. Alzheimer Disease – Effect of Continuous Intracerebroventricular Treatment with GM1 Ganglioside and a Systematic Activation Programme. Dement Geriatr Cogn Disord. 2002;14(3):128-36.
- Schneider JS, Roeltgen DP, Mancall EL, Chapas-Crilly J, Rothblat DS, Tatarian GT. Parkinson’s disease: improved function with GM1 ganglioside treatment in a randomized placebo-controlled study. Neurology. 1998;50(6):1630-6.