An international clinical trial has found that a new drug for Huntington’s disease is safe, and that treatment with the drug successfully lowers levels of the abnormal protein that causes the debilitating disease in patients.
In the 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 lowers 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.”
Leavitt, who is also a senior scientist at the Centre for Molecular Medicine and Therapeutics in the UBC faculty of medicine, treated all the Canadian participants in this study, including the first patient enrolled in the study in September 2015.
Huntington disease (HD) is a fatal genetic neurological disease. It usually develops in adulthood and causes abnormal involuntary movements, psychiatric symptoms and dementia. About one in 10,000 people in Canada have HD. To date, no effective treatments have been proven to slow down progression of this disorder. HD is caused by a single known genetic mutation, and each child of a carrier of the mutation has a 50 per cent chance of inheriting the disease.
Read more at the University of British Columbia