Cell therapy repairs multiple sclerosis damage and restores motor functions in mice

Glial cells play several key roles in the central nervous system, including supplying oxygen to neurons and forming myelin—the protective, fatty substance that protects the nerve cells’ axons. In multiple sclerosis (MS), glial cells called oligodendrocytes are attacked by the immune system, causing a breakdown of myelin that disrupts the signals between nerve cells and results in a loss of motor and sensory functions.

A team of researchers at the University of Rochester Medical Center (URMC) is developing a method for regenerating myelin with progenitor glial cells. When they transplanted the cells into mouse models of MS, the cells transformed into new oligodendrocytes and restored myelin. They reported the results of the study in the journal Cell Reports.

Now, a company that was spun out of the university last year, Oscine Therapeutics, is preparing the cell therapy for human clinical trials in MS and other glial diseases, according to a statement. Oscine received an undisclosed amount of funding last year from Sana Biotechnology, a cell therapy developer backed by funding from Arch, Flagship and F-Prime.

The cell therapy got its start in the lab of Steve Goldman, M.D., Ph.D., professor of neurology and neuroscience at URMC, who led a 15-year effort to figure out how to coax stem cells into becoming glial cells. Their hope was to develop the therapy to treat progressive multiple sclerosis—a stage of the disease during which oligodendrocytes become exhausted and can no longer make myelin.

In the mouse study, Goldman and colleagues showed that after transplantation, the human glial progenitor cells migrated to damaged areas of the brain. After they created new oligodendrocytes, myelation was restored, as was motor functioning, the team reported. Goldman is the scientific founder and a shareholder in Oscine.

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Much of the regenerative medicine research in MS is focused on restoring myelin, and several different approaches are under investigation. Last year, researchers from VA Maryland Health Care System and the University of Maryland reported that when they implanted stem cells with the surface protein CD34 into mouse models of MS, the cells grew into myelin-forming glial cells. Other experimental approaches to regenerating myelin include using microRNAs and reprogrammed skin cells.

San Francisco-based Atara Biotherapeutics is targeting Epstein-Barr virus (EBV), which has been shown to raise the risk of MS. The company is developing T-cell therapies targeting EBV and has started early human trials. Last year, Atara released data from a phase 1 study showing a partial improvement in MS symptoms six months following treatment with one of its EBV-targeted therapies, ATA188.

The FDA is currently reviewing Oscine’s plan to bring URMC’s glial cell therapy into clinical trials, according to the statement. The company’s researchers believe it could prove promising in other diseases marked by glial defects, too, including Huntington’s.