Giving Huntington’s disease the one-two punch

Tuesday, July 26, 2016 - 10:31 in Biology & Nature

A major, multi-institutional study based at Harvard-affiliated Massachusetts General Hospital (MGH) has identified a promising treatment strategy for Huntington’s disease (HD). The team’s identification of a novel compound, MIND4, appears to protect against neurodegeneration in cellular and animal models of HD by means of two separate mechanisms — inhibiting a regulatory enzyme of the nervous system (SIRT2), and stimulating activity of the NRF2 pathway, which regulates the expression of protective, antioxidant proteins. The report will be published online in the journal Cell Chemical Biology. “Based on numerous studies, it has become evident that the pathologies of neurodegenerative diseases, including Huntington’s disease, are very complex, so targeting multiple pathways may help us achieve maximum therapeutic benefit,” said Aleksey Kazantsev, who led the study as an investigator at the MassGeneral Institute for Neurodegenerative Disease (MIND). “The lead compound identified in the current study has two distinct mechanisms, both of which are shown to be...

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