A small molecule drug has shown promise as a potential new treatment for multiple sclerosis (MS), a team of researchers has said. The results are published in the journal Science Advances.
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.
It is known that MS damages myelin, a protective sheath that forms around nerves in the brain and spinal cord. As the myelin damage is triggered by inflammation in the immune system, up until now all current drug treatments for MS target the immune system.
In the study, senior scientist Dr. Fang Liu and her team treated MS in a completely different way—targeting the glutamate system. Study results showed that the newly synthesized lead compound not only reduced MS-like symptoms, it also may repair the damaged myelin in two different pre-clinical models of MS.
“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 Dr. 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.”
Dr. Iain Greig, Reader in Medicinal Chemistry at the University of Aberdeen, alongside his team, are working to turn 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.
