“This trial offers our patients hope in slowing down or even stopping the progression of this devastating disease,” said Dr. Tiago MestreThe Ottawa Hospital’s Parkinson’s Disease and Movement Disorders clinic is bringing new hope to people with Huntington’s disease (HD) through an innovative clinical trial of a gene-targeted therapy. HD is an inherited disorder that often begins with subtle mood and movement problems between the ages of 30 and 50 and eventually progresses to severe dementia and premature death.

It is caused by a genetic mutation in the huntingtin gene, which results in a defective and toxic protein. The trial is testing a promising approach called RNA interference, which targets, or ‘silences,’ a specific genetic sequence to prevent the production of the toxic huntingtin protein.

“This trial offers our patients hope in slowing down or even stopping the progression of this devastating disease,” said Dr. Tiago Mestre, neurologist, senior scientist and study lead at TOH and associate professor at the University of Ottawa. “It is so important that The Ottawa Hospital has the capacity to allow us to bring innovative trials to our community.”

The first-in-human Phase 1 trial, sponsored by Alnylam Pharmaceuticals, is primarily assessing safety and target engagement. The Ottawa Hospital is one of 16 participating sites around the world—and was among the first to take part, having enrolled a patient in the initial cohort of six participants.

Funding: Alnylam Pharmaceuticals

The Ottawa Hospital is a leading academic health, research and learning hospital proudly affiliated with the University of Ottawa and supported by The Ottawa Hospital Foundation.