Multiple Sclerosis Symptoms Reduced with Experimental Cancer Drug

In mouse models of multiple sclerosis (MS), experimental cancer drugs have shown therapeutic promise, according to findings published in the journal Nature.

Researchers from NYU Langone Medical Center aimed to characterize the effect on remyelination in adult neural stem cells in mice models using the experimental cancer drug called GANT61 over a six year period. The drug is designed to block the ability of protein Gli1, which is a part of the “sonic hedgehog signaling” process. That biological pathway closely tied to neural stem cells and can affect the growth of some cancers. The signaling was heightened in tissue samples taken from brain lesions in human MS patients.

The mice used in the study were administered GANT61 daily for one month while other, non medicated mice served as the control subjects. The drugged mice showed 50 percent more myelin at the end of the observation period when compared to the non medicated mice, the researchers found. Additionally, the GANT61 treated mice demonstrated an eightfold increase in the number of neural stem cells that had moved to the myelin damaged areas of the mice’s brain, when compared to the non medicated mice. Those cells eventually developed into myelin producing oligodendrocytes, the researchers said, and added that the untreated mice did not display this increased cell count.

Another interesting finding the researchers highlighted was that the treated mice coped well and recovered from MS like paralysis and leg weaknesses, while the untreated mice suffered from leg and bladder weaknesses. Those symptoms are frequently experienced by MS patients.

The researchers pointed out that the findings are the first of their kind to demonstrate that drugs targeted at sonic hedgehog can treat neurological disorders like MS even though the pathway was first discovered in the 1980s. In MS, targeting the sonic hedgehog pathway is different than blocking the entire pathway, the researchers said, which other experiments have done. In those studies, there was no remyelination and oligodendrocyte maturation was stopped.

“Our study results suggest that a potential long term strategy for treating MS would involve treatments that separately target both neural stem cells, to help turn them into mature oligodendrocytes, as well as young and immature oligodendrocytes to produce more myelin,” explained senior investigator James Salzer, MD, PhD, in a press release. “Our findings also make clear that there is a resident population of adult neural stem cells that we can target and recruit to treat the disorder.”

These findings can likely contribute to pinpointing the underlying causes and symptoms of MS and develop better treatments for the disease.