Experimental compound boosts myelin repair in preclinical MS study

A small molecule called CN045 may help repair myelin — the protective coating around nerve fibers that is damaged in multiple sclerosis (MS) — according to a new preclinical study.

Researchers found that CN045 promoted the maturation of myelin-making cells in lab tests and modestly increased remyelination, the process of repairing damaged myelin, in a mouse model. While much more research is needed to optimize the compound before it could be considered for testing in people, the findings support CN045 as an early lead candidate for developing a remyelination therapy for MS, the researchers said.

The study, “A novel small molecule remyelination therapy for multiple sclerosis,” was published in npj Drug Discovery. The work was supported by the National Institutes of Health, the National Multiple Sclerosis Society, and Ohio Fast Forward.

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Study points to new target for protecting myelin in multiple sclerosis

Why repairing myelin matters in MS

In MS, the immune system mistakenly attacks myelin, the fatty coating that insulates nerve fibers and helps electrical signals travel efficiently through the brain and spinal cord, much like rubber insulation around a copper wire. As myelin is lost, nerve signals are disrupted, contributing to MS symptoms.

More than 20 disease-modifying therapies are approved for MS. These treatments suppress the inflammatory immune activity that damages myelin, reducing relapses and new lesions, but they do not directly repair existing myelin damage. In theory, a therapy capable of restoring myelin might help protect nerve fibers and preserve nerve function.

Within the brain and spinal cord, myelin is produced by cells called oligodendrocytes, which develop from immature oligodendrocyte progenitor cells (OPCs). Encouraging OPCs to mature into myelin-producing cells has long been considered a promising strategy for repairing damaged myelin in MS.

To identify compounds that could do this, a team led by researchers in the U.S. screened 20,000 molecules selected for properties favorable to use in the central nervous system for their ability to promote OPC maturation. CN045 emerged as the lead candidate for further study.

In lab experiments using human OPCs, treatment with CN045 increased the number of mature, myelin-producing oligodendrocytes. The newly formed cells also formed myelin-like coverings around nanofibers designed to mimic nerve fibers, showing that they could carry out a key function of mature oligodendrocytes.

CN045 also promoted modest remyelination in a mouse model of myelin loss, indicating the compound can promote myelin repair in living animals.

CN045 shows promise but needs further optimization

The researchers also found several characteristics that could make CN045 a good drug candidate. The molecule was able to enter the brain, showed low cell toxicity in laboratory and animal testing, and had an intellectual-property path that the researchers said appeared open, potentially easing future development.

One drawback of CN045 is that it is rapidly broken down in the body, reducing how long it remains active. However, the researchers said its chemical structure is well-suited for modifications aimed at improving its stability and other drug-like properties, although further study is needed.

Overall, the team said CN045 represents a promising starting point to develop future remyelination therapies.

“CN045 is brain-penetrable with low [cell toxicity] … and appears to be an attractive contender that can be improved by further lead optimization to generate a clinical candidate for enhancing remyelination in MS patients,” the team concluded.