Neural stem cell therapy repairs myelin in MS mouse model: Study

Research suggests potential new way to treat progressive forms of disease

by Steve Bryson, PhD | July 11, 2025

Treatment with neural stem cells, which have the ability to differentiate into neurons and other supportive cells of the nervous system, was safe and significantly boosted myelin repair in spinal cord lesions in a mouse model of multiple sclerosis (MS), a study showed.

The stem cells were able to differentiate into fully mature oligodendrocytes, the cells responsible for producing myelin, and to directly drive myelin formation, even in regions where myelin repair was chronically impaired.

“This research provides critical evidence that induced neural stem cell grafts can effectively turn into myelin-producing cells within the damaged [brain and spinal cord], suggesting a potential new way to treat progressive MS,” Luca Peruzzotti-Jametti, MD, PhD, a scientist at the University of Cambridge in the U.K. and the study’s first author, said in a university news story.

The study, “Remyelination of chronic demyelinated lesions with directly induced neural stem cells,” was published in the journal Brain.

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Few treatment options for progressive forms of MS

MS is caused by an immune-mediated attack on the myelin sheath, an insulating layer surrounding nerve fibers that speed the transmission of electrical impulses. The resulting inflammation further damages nerve cells and oligodendrocytes.

Although there are several disease-modifying therapies approved to treat MS, these mostly work to reduce inflammation and lower the rate of relapses. Their ability to slow disability progression, however, is limited, so there are very few options for progressive forms of MS.

Neural stem cells, or NSCs, are specialized cells that can differentiate into nerve cells and other types of cells within the brain and spinal cord. They can also release signaling molecules with potent anti-inflammatory effects.

Recent Phase 1 trials have supported the safety and feasibility of NSC transplantation in people with progressive MS, while showing the approach can reduce markers of inflammation and nerve damage and slow disability progression.

“However, the mechanisms by which NSC grafts could promote [brain and spinal cord] remyelination need to be carefully assessed before their widespread clinical adoption,” the researchers wrote.

Neural stem cell treatment promoted faster resolution of inflammation

To learn more, the team examined whether NSCs can promote new myelin production in a mouse model of MS. Mice were treated with a chemical that induced demyelinating lesions in the spinal cords of healthy mice, and were randomly assigned to receive NSCs or a vehicle as a control.

Tissue examination confirmed NSCs successfully integrated into the demyelinated spinal cord and induced myelin repair. Taking a closer look, the team found that the transplanted cells differentiated into mature oligodendrocytes and also caused the mice’s oligodendrocyte progenitor cells to mature into myelin-producing oligodendrocytes. NSCs also promoted a faster resolution of inflammatory responses.

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