An experimental drug originally identified in a National Cancer Institute library of chemical compounds as a potential therapy for brain and basal cell cancers improves the symptoms of mice with a form of the debilitating neurological disorder multiple sclerosis (MS), according to new research from NYU Langone Medical Center.
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.
A report describing the findings is being published in the journal Nature online Sept. 30.
In the study, mice with chemically damaged brain myelin were given daily doses of GANT61 for one month. Results showed that mice that 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.