Research Focuses on New Drug Targets for Regenerative MS Therapies

In a groundbreaking new study, researchers have identified a molecular pathway for stimulating the regeneration of new myelinating cells, and repairing the damage of debilitating neurological diseases. According to a study authors, the findings could pave the way for potential new regenerative therapy candidates for neurological conditions, such as multiple sclerosis (MS).

The degeneration of myelin—the protective covering that surrounds nerve cells in the brain and spinal cord—causes disease progression in MS. Previous research has identified several small molecules that promote myelin repair in mice by enhancing the conversion of a type of stem cell called oligodendrocyte progenitor cells (OPCs) to oligodendrocytes.

“Many labs, including at Case Western Reserve, had identified drug candidates that kickstart the formation of new myelin, but exactly how each of these molecules affected brain cell function wasn’t clear,” study author Drew Adams, PhD, professor of novel therapeutics and assistant professor of genetics and genome sciences at Case Western Reserve University School of Medicine, said in a press release.

In the study, researchers from Case Western Reserve University found that many of the previously identified molecules trigger myelin regeneration by blocking specific enzymes, known as CYP51, TM7SF2, or EBP, which are used by brain cells to produce cholesterol.

Miconazole, a drug typically used to treat athlete’s foot, enhances myelin formation by inhibiting these cholesterol-producing enzymes, according to the study. The team found 20 additional therapies that also enhance myelin formation in this way. The researchers were able to pinpoint which enzymes were being blocked by which drugs from the accumulation of metabolites in the cholesterol pathway.

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