MassGeneral Institute for Neurodegererative Disorders (MIND) researchers have identified a compound that may lead to a treatment that could protect against the effects of Huntington's Disease (HD).
Their report, which will appear in the Proceedings of the National Academy of Sciences, describes how a small molecule called C2-8 appears to delay the loss of motor control and reduce neurological damage in a mouse model of the disorder. The study is receiving early online release.
“We found that C2-8 slows the progress of HD in a mouse model and might do the same thing in human patients, if it or its biochemical relatives can be translated into a drug,” says Steven Hersch, MD, PhD, of MIND and the Massachusetts General Hospital (MGH) Department of Neurology, who led the study. “What we don't know yet is precisely how it works, what molecules it interacts with in cells and how potent it might be.”
C2-8 was first identified as a candidate treatment for HD by MIND researcher Aleksey Kazantsev, PhD, based on its ability to block the aggregation of the mutant huntingtin protein in yeast and animal tissue and to improve function in a fruit fly model. The current study was designed to further investigate its potential as a therapeutic drug. The researchers first confirmed that oral doses of C2-8 can cross the blood-brain barrier and are nontoxic in a mouse model of HD. They also found that C2-8 does not interact with a number of molecules predictive of negative side effects.