Huntington drug successfully lowers levels of disease-causing protein

An international clinical trial has found that a new drug for Huntington disease is safe, and that treatment with the drug successfully lowers levels of the abnormal protein that causes the debilitating disease in patients. In a study published today in the New England Journal of Medicine, researchers from UBC and their colleagues have demonstrated for the first time that the drug, IONIS-HTTRX (now known as RO7234292) successfully lowered levels of the mutant huntingtin protein—the toxic protein that causes Huntington disease—in the central nervous system of patients. "This is a tremendously exciting and promising result for patients and families affected by this devastating genetic brain disorder," said Dr. Blair Leavitt, neurologist and director of research at the Centre for Huntington Disease at UBC. "For the first time, we have evidence that a treatment can not only decrease levels of the toxic disease-causing protein in patients, but that it is also safe and very well tolerated."