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Experimental drug may spur myelin repair

10/2/2015

An experimental drug originally identified as a potential therapy for brain and basal cell cancers improves the symptoms of mice with the mouse model of multiple sclerosis, according to new research from NYU Langone Medical Center. The experimental drug, GANT61, blocks the action of a key protein that is involved in so-called sonic hedgehog signaling, whose signaling is raised in tissue samples taken from brain lesions in patients with MS.

In the study, mice with chemically damaged brain myelin were given daily doses of GANT61 for one month. Mice that received the drug had 50 percent more myelin at the end of treatment than did untreated mice. Moreover, researchers found that the GANT61-treated mice had an eightfold increase in the number of neural stem cells that migrated to myelin-damaged areas of the brain and eventually developed into myelin-producing oligodendrocytes. They were also able to recover from an initial bout of MS-like paralysis and leg weakness. The authors say the research is the first strong evidence that in MS, targeting part of the sonic hedgehog pathway has a fundamentally different effect than blocking the entire pathway, which in other experiments elsewhere did not produce any remyelination but instead halted oligodendrocyte maturation.

The experiments took six years to complete and are believed to be the first to demonstrate that neural stem cells, and not just early forms of oligodendrocytic cells, can be modified and recruited into myelin repair. Results of mouse model studies sometimes do not translate to humans and may be years away from being a marketable treatment.

"Our study results suggest that a potential long-term strategy for treating multiple sclerosis 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. 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," according to senior study investigator Dr. James Salzer.

The findings were published in the journal Nature.



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