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Innovative MS Research

11 December 2023

Researchers in Canada have developed a small molecule drug that shows potential as a new treatment for multiple sclerosis (MS). This research focuses on targeting the glutamate system rather than the immune system, which differs from existing MS therapies.

It is known that MS damages myelin, a protective sheath that forms around nerves in the brain and spinal cord. Because myelin damage is triggered by inflammation in the immune system, up until now all current drug treatments for MS target the immune system.

But a new study at CAMH in Toronto treated MS in a completely different way by targeting the glutamate system. Glutamate is an amino acid that acts as a neurotransmitter in the brain, triggering other neurons to release their own chemical messages to result in something happening. For example, if glutamate triggers a motor neuron, it might cause a muscle to contract.

Results from the study showed that the newly synthesized lead compound not only reduced MS-like symptoms, it may also repair the damaged myelin. This represents a key advancement and a significant leap toward clinical trials, offering new hope for MS patients.

“Our compound had a stunning effect on rescuing myelin and motor function in the lab models, and I hope these effects will translate to the clinic to add to current treatments and bring new hope to patients with MS,” said lead researcher, Dr Fang Liu. “As with cancer chemotherapy drug cocktails, simultaneous targeting of the MS disease pathway at multiple points can have synergistic effects and result in better outcomes.”

A team at the University of Aberdeen, led by Dr Iain Greig, is now working to turn the molecules identified by Dr Liu into advanced drug-like molecules suitable for continued development towards clinical use in patients. They believe that the evidence of efficacy and tolerability generated in this study make it a good candidate for human trials. Dr Greig said: “In all my years as a medicinal chemist, I have never seen a more promising starting point for a drug development project.”

The next steps will involve further pre-clinical research, including investigating safety and stability of the compound. CAMH and the University of Aberdeen have already filed patent applications to protect this research and are actively working to further advance this work towards clinical trials over the next few years.

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