Preclinical studies conducted by CAMH using a small molecule drug have shown promise as a potential new treatment for multiple sclerosis (SM). Expanding on Dr. Fang Liu’s previous work identifying a new drug target for the treatment of MS, she and her team have now created a small molecule compound that is effective in two different animal models of MS.
This represents a key advance that brings multiple sclerosis research closer to the clinic to impact patient care.
The results of the study were published in the journal Science Advances.
Multiple sclerosis: some details on the new research
MS is a progressive neurological disease that currently has no cure. It is associated with a wide range of debilitating symptoms, including problems with coordination, cognition, muscle weakness, and depression. For unknown reasons, it is more common in northern latitudes and more than twice as common in women.
Multiple sclerosis is known to damage myelin, a protective sheath that forms around nerves in the brain and spinal cord. Because myelin damage is triggered by inflammation of the immune system, so far all current drug treatments for MS target the immune system.
In this study, CAMH senior scientist Dr. Fang Liu and her team treated multiple sclerosis in a completely different way by targeting the glutamate system. The study results showed that the newly synthesized lead compound not only reduced MS-like symptoms, but can also repair damaged myelin in two different preclinical models of MS.
“Our compound had a surprising effect on rescuing myelin and motor function in laboratory models, and I hope these effects translate into the clinic to add to current treatments and bring new hope to MS patients,” said Dr. Liu. “As with cancer chemotherapy drug cocktails, targeting the MS disease pathway at multiple points simultaneously can have synergistic effects and lead to better outcomes.”
Dr Iain Greig, Reader in Medicinal Chemistry at the University of Aberdeen, together with his team, are working to transform the molecules identified by Dr Liu into advanced ‘drug-like’ molecules suitable for continued development towards clinical use in patients.
He added: “In all my years as a medicinal chemist, I have never seen a more promising starting point for a drug development project. It has been a tremendous pleasure to be involved in this program and I look forward to continuing to lead it.” towards the clinic.”
“We are pleased to have helped enable the initial development of a new neuroprotective strategy for MS, and we look forward to seeing it progress through the crucial next steps needed to determine its potential benefits for people living with MS.” , said Walt Kostich, Ph. D., head of the National MS Society (USA) Fast Forward commercial research program.
Dr. Liu believes that the efficacy and tolerability evidence generated in this study for the small molecule drug makes it a good candidate to be developed for human testing. The next steps in the drug’s development will involve further preclinical research, including studying the safety and stability of the compound.
CAMH and the University of Aberdeen have already filed patent applications to protect this research and are actively seeking industrial partners to further advance this work towards clinical trials in the coming years.
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