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An Experimental Cancer Drug Reduces MS Symptoms

November 19, 2015

A study published in the journal Nature states that in mouse models of MS, experimental cancer drugs have shown great therapeutic promise. Researchers from NYU Langone Medical Center aimed to characterize the effect on remyelination in adult neural stem cells in mice models using an experimental cancer drug called GANT61.

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 were administered the drug daily for one month while the non-medicated mice served as the control group. At the end of the observation period, the mice who were taking the drug showed 50% more myelin, as compared to the control group. Also, the medicated mice showed an eightfold increase in the number of neural stem cells that had moved to the damaged areas of the mice’s brain. The medicated mice were able to overcome “MS-like” paralysis and leg weakness.

“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.”