Stem cell therapy involves the use of stem cells to treat or prevent a medical condition. There are now many potential uses from healing brain and spinal cord injuries to overcoming certain forms of blindness, but transplanting bone marrow — in order to treat cancers such as leukemia and lymphoma — is the only form of stem cell therapy widely practiced today.

Multiple sclerosis, MS, is an inflammatory autoimmune disease that causes damage to the nervous system and can result in a wide range of physical, mental, and psychiatric symptoms. Furthermore, rates for this disabling condition appear to be increasing.

The mice began to regain motor skills after only 10 to 14 days after treatment. Six months later, they showed no signs of slowing down.

Exciting new research has yielded spectacular results that suggest stem cell therapy may also be useful in treating multiple sclerosis (MS).

In less than two weeks following treatment with human neural stem cells, disabled mice with the symptoms of MS were able to walk. Prior to the stem cell transplantation, the mice required hand-feeding because they could not stand long enough to eat or drink water on their own.

What began as a routine experiment with researchers suspecting that the mice would very likely reject the transplanted cells, in a manner similar to the way some patients reject organ transplants, instead produced a stunning result.

“My postdoctoral fellow Dr. Lu Chen came to me and said, ‘The mice are walking.’ I didn’t believe her,” recalled Tom Lane, Ph.D., a faculty member at the University of Utah and co-author of the paper.

The mice began to regain motor skills as soon as 10 to 14 days after treatment. Six months later, they showed no signs of slowing down.

Current medications for MS act by slowing progression of the disease by preventing the immune system from targeting and damaging the nervous system.

The results of this study offer hope for finding more effective treatments capable of halting or reversing MS, thereby helping patients in the later stages of the disease for whom there are no treatments.

While the researchers' expectation that the transplanted stem cells would be rejected eventually proved accurate, they survived long enough to send chemical signals prompting repair of the mouse’s own cells that had been damaged by MS. This realization has important implications for translating the work into clinical trials.

“Rather than having to engraft stem cells into a patient, which can be challenging from a medical standpoint, we might be able to develop a drug that can be used to deliver the therapy much more easily,” said Lane, commenting on the chemical signal responsible for the repair.

First, however, the researchers must assess the durability and safety of the therapy in mice. “We want to try to move as quickly and carefully as possible,” said Lane, who hopes the work will lead to advances that could ease the burden associated with MS, a disease that affects more than 2.3 million people globally.

The findings are published in Stem Cell Reports.