Posted: August 2, 2014
Researchers at the University of Alberta have found that ganglioside metabolism is impaired in Huntington’s disease (HD) and that administration of GM1 restores normal ganglioside levels and decreases HD cell susceptibility to apoptosis. Gangliosides are sialic acid-containing glycosphingolipids that are particularly enriched in the brain and play an essential role in cell signalling. Levels of GM1, the most abundant ganglioside in the brain, were found to be reduced in HD cell models and in fibroblasts from HD patients.
HD is a genetic neurodegenerative disorder characterized by involuntary movements and decline of motor, cognitive and psychiatric functions. There are very few treatment options available for HD and they are limited to the management of associated symptoms only. There is, therefore, a great need for therapeutics specifically addressing the pathology of the disease.
- GM1 has been shown to be safe and well tolerated in clinical trials for other neurological indications.
- Candidate for orphan drug approval.
This technology would be of particular interest to companies interested in developing treatments for neurodegenerative diseases such as HD.
Technology Management Group
TEC Edmonton – University of Alberta