Therapy candidates that block enzymes responsible for making cholesterol can promote myelin regeneration, a discovery that could lead to new regenerative medicines capable of treating multiple sclerosis (MS) and other neurological diseases, according to a study.
In fact, Convelo Therapeutics plans to do just that, announcing its intention to use the breakthrough research as the foundation for its new drug discovery platform, according to a company press release.
The study, “Accumulation of 8,9-unsaturated sterols drives oligodendrocyte formation and remyelination,” was published in the journal Nature.
MS is a chronic progressive disease caused by the rapid breakdown and insufficient regeneration of myelin — the protective covering surrounding nerve cells in the brain and spinal cord.
“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,” Drew Adams, PhD, assistant professor of genetics and genome sciences at Case Western Reserve University School of Medicine, said in press release from the university.
A type of stem cell — called oligodendrocyte progenitor cells (OPCs) — develops into oligodendrocytes, the cells responsible for the regeneration of myelin. Previous studies have identified several small molecules that promote myelin regeneration in mice by enhancing the conversion of OPCs to oligodendrocytes.
Now a research team led by scientists from Case Western Reserve University has found that many of these small molecules trigger the development of oligodendrocytes and myelin regeneration by blocking specific enzymes — namely CYP51, TM7SF2, or EBP — needed for the production of cholesterol.
Blocking these enzymes results in the buildup of a molecule called 8,9-unsaturated sterol, which acts as a trigger for the conversion of OPCs to oligodendrocytes, the team reported.
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