AANS Neurosurgeon | Volume 26, Number 1, 2017

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Experimental Cancer Drug Shows Therapeutic Promise in Mouse Models of MS

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According to researchers from NYU Langone Medical Center, an experimental drug originally identified in a National Cancer Institute library of chemical compounds as a potential therapy for brain and basal cell cancers has shown to improve the symptoms of mice with a form of the debilitating neurological disorder multiple sclerosis (MS). The experimental drug employed by the NYU Langone team of neuroscientists is called GANT61. It blocks the action of a key protein, Gli1, which is involved in so-called “sonic hedgehog” signaling, a biological pathway closely tied to neural stem cell development and the growth of some cancers, and whose signaling is raised in tissue samples taken from brain lesions in patients with MS. During the study, mice with chemically-damaged brain-myelin were given daily doses of GANT61 for one month. Results showed that mice who received the drug had 50-percent more myelin at the end of treatment than did untreated mice. Myelin is the nerve-protecting sheath whose degradation is a principal cause of MS. Moreover, the researchers say, they 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. Untreated mice did not show this increase. According to the study’s senior investigator, the experiments, which took six years to complete, are believed to be the first to demonstrate that neural stem cells, not just early forms of oligodendrocytic cells, can be modified and recruited into myelin repair. To read more about this study, click here.

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