There is new hope for thousands of people diagnosed with Huntington Disease, as the results of a clinical drug trial with Canadian patients were announced on Monday.
Huntington has been described as Alzheimer’s, Parkinson’s and ALS all rolled into one debilitating condition. It attacks the brain, and can affect a range of abilities, from motor skills to one’s connection to reality.
“It basically means that parts of their brain really stop working,” says Dr. Rachel Harding, a biomedical scientist at the University of Toronto. “It’s really quite a horrific disease.”
The news has been more than 10 years in the making, with 46 patients in the UK, Germany and Canada in the initial trial.
“I am cautiously optimistic that this could not only halt the progression of the disorder, but lead to an improvement in symptoms,” says Dr. Blair Leavitt, research director at the Centre for Huntington Disease at the University of British Columbia.
The very first patient was treated in Vancouver in September 2015.
“This is the first time that a treatment in people has been shown to be safe and has shown that it alters the levels of abnormal proteins that causes Huntington Disease,” says Leavitt, who is one of the international researchers involved.
Those with the disease carry a mutation in the huntingtin gene, which results in the production of a toxic protein that attacks the brain and nervous system.
“There are not many good days when you have a neurodegenerative disorder,” says Dr. Karim Nader, a neuroscientist at McGill University. He also suffers from Huntington Disease, which has affected his motor skills and speech.
“It effects every aspect of our lives,” says Nader. “Each time you try to board a plane, to come back to a conference or go to a conference, each time the border agents are convinced that you must have been drinking.”
Huntington is a genetic disease — children who have a parent with Huntington’s have a 50/50 chance of inheriting it. The consequences are also fatal, with many dying early from a list of complications including pneumonia and heart failure.
“We’ve adanced into a new era,” says Leavitt. “We’re now using all of the information that we’ve gathered over the last 20 years, and really targeting the causes of this disease.”
The next step is phase three of the trial. Researchers hope it will prove the treatment, which is an injection into the spine, can stop the disease in its tracks.
Though there’s no word on when the next stage will begin in Canada, CityNews is told it will be announced in the very near future. When it gets started, Nader says he’ll be taking part.
“It’s a blessing,” says Nader. “If you believe in evolution, you wouldn’t say it’s a miracle, so let’s say it’s a genetic miracle.”