# Characterizing the oxysterol, 20-hydroxycholesterol, as a mediator of remyelination in multiple sclerosis

> **NIH NIH R21** · DUKE UNIVERSITY · 2021 · $442,750

## Abstract

Multiple sclerosis (MS) is the most common neurological disease of young adulthood, affecting an estimated 1
million individuals in the U.S. and 2.5 million worldwide. MS is an autoimmune disease mediated by immune
cells that trigger demyelination and neuronal damage of the central nervous system (CNS), resulting in
debilitating neurological symptoms. While disease-modifying therapies have proven to be efficacious, they only
prolong remission, they do not change disease course, and the majority of individuals with MS will likely
experience worsening of clinical symptoms during the course of their disease. There is a significant gap in
knowledge with respect to curative therapies for MS that prompt oligodendrocyte precursor cells to differentiate
into mature oligodendrocytes (ODs), the main remyelinating cells within the adult CNS. Presented are exciting
preliminary data in a white matter injury model of adult mice that establishes that 20-hydroxycholestrol (20HC)
is capable of triggering remyelination in the CNS, and that it is capable of differentiating new ODs from the
quiescent pool of OPCs in the CNS beyond the limited spontaneous regeneration that occurs during disease
course. This resubmitted proposal builds upon this evocative preliminary data in an animal model of
demyelination and proposes the application of leading edge molecular approaches to understanding the
mechanisms of 20HC effect. The long-term goal of this proposal is to identify the efficacy of 20HC as a completely
novel drug for reversing the progressive course of MS.

## Key facts

- **NIH application ID:** 10311395
- **Project number:** 1R21NS117020-01A1
- **Recipient organization:** DUKE UNIVERSITY
- **Principal Investigator:** Eric J Benner
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $442,750
- **Award type:** 1
- **Project period:** 2021-07-01 → 2024-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10311395

## Citation

> US National Institutes of Health, RePORTER application 10311395, Characterizing the oxysterol, 20-hydroxycholesterol, as a mediator of remyelination in multiple sclerosis (1R21NS117020-01A1). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10311395. Licensed CC0.

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