Pre-clinical studies led by CAMH have unveiled a potential breakthrough in multiple sclerosis (MS) treatment, using a small molecule drug. Published in Science Advances, this research, building on Dr. Fang Liu's earlier findings, introduces a compound effective in two MS animal models, marking a significant stride toward clinical application.
MS, an incurable progressive neurological disease, manifests debilitating symptoms such as coordination issues, cognitive impairment, muscle weakness, and depression. While the exact cause remains unknown, MS damages myelin, the protective sheath around nerves, triggered by inflammation. Existing treatments target the immune system, but Dr. Liu's team diverged, focusing on the glutamate system.
The synthesized lead compound not only alleviated MS-like symptoms but also exhibited potential myelin repair in pre-clinical models. Dr. Liu envisions its translation to the clinic, complementing current treatments and offering hope to MS patients. Targeting MS at multiple points, akin to cancer chemotherapy cocktails, could yield synergistic effects and improved outcomes.
Dr. Iain Greig from the University of Aberdeen is collaborating to refine Dr. Liu's molecules for clinical use. Expressing optimism, he deems it an unparalleled starting point for drug development, anticipating further progress toward clinical applications.
Walt Kostich, head of the National MS Society's Fast Forward research program, acknowledges the contribution to a novel neuroprotective strategy for MS, anticipating its progression through critical stages. Dr. Liu, confident in the drug's efficacy and tolerability, deems it a strong candidate for human trials. Further pre-clinical research, including safety and stability assessments, will precede potential clinical trials.
CAMH and the University of Aberdeen have safeguarded this research through patent applications, actively seeking industry partnerships for advancing toward clinical trials in the coming years. The collaborative efforts signify a promising avenue for reshaping MS treatment paradigms.