# Discovering new therapies to promote myelin repair

> **NIH NIH R21** · UNIVERSITY OF VIRGINIA · 2021 · $440,030

## Abstract

Abstract:
Multiple sclerosis (MS) is a neurodegenerative, autoimmune disease characterized by the destruction of the
neuron-surrounding myelin sheath. Myelin has the double duty of assisting with the propagation of nerve
impulses and shielding neurons from external harm. Once immune cells strip the neuron of myelin, exposed
axons begin to decay, leading to the debilitating symptoms of MS. Currently approved therapies for MS are
aimed at inhibiting the immune response and do not address the need to promote myelin repair. Development
of therapeutics that would activate myelination and prevent the irreversible changes associated with neuronal
death is paramount to improving the quality of life and survival of MS patients. The central nervous system (CNS)
contains oligodendrocyte precursor cells (OPCs) that have the potential to differentiate into mature
oligodendrocytes and remyelinate denuded axons, a process that is affected in MS patients. Our long-term goal
is to develop new MS treatments that halts neurodegeneration by promoting myelin repair, affording a potentially
curative drug. The innovation driving this proposal is an in silico screen that allowed discovery of a molecule
that drives OPC differentiation in vitro. The efficacy of this compound was validated in a mouse model of
experimental autoimmune encephalomyelitis (EAE, an animal model of MS). Our goal in this proposal is to shed
light on the novel mechanism of action of this agent and identify chemotypes that activate remyelination. Guided
by strong preliminary evidence, this goal will be achieved by pursuing two specific aims: 1) discover the
receptor/protein target of this agent, which is key to OPCs differentiation, and 2) develop our hit compound into
drug-like molecules with efficacy and potency suitable for testing in animal models of MS. Our approach is
significant because it will lead to identification of protein targets in MS that promote myelination and new
therapeutics with novel modes of action. Our investigations will lead to fundamental advances in understanding
the regulation of myelination.

## Key facts

- **NIH application ID:** 10314338
- **Project number:** 1R21NS124168-01
- **Recipient organization:** UNIVERSITY OF VIRGINIA
- **Principal Investigator:** Alban P Gaultier
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $440,030
- **Award type:** 1
- **Project period:** 2021-08-01 → 2024-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10314338

## Citation

> US National Institutes of Health, RePORTER application 10314338, Discovering new therapies to promote myelin repair (1R21NS124168-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10314338. Licensed CC0.

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