Potential ALS treatment to be studied in Montreal

JOver a decade after the ice bucket challenge went viral, research in Montreal hopes to slow the pace of ALS. (Photo AP/Walla Walla Union-Bulletin, Greg Lehman, Archives) (Greg Lehman/The Associated Press)

This study follows up on preclinical work carried out by Professor Richard Robitaille at the Université de Montréal, in which the treatment improved the strength and mobility of the mice to which it was administered.

Dr. Olivier Blanchard and his team want to find out whether a drug already approved for overactive bladder could block the overactive cells that inevitably end up damaging the connection between the nerves and muscles of ALS patients.

“We see very early in the process (of the disease) that the junction between the nerve and the muscle is very diseased,” said Blanchard. “And not only is the neuromuscular junction not good, but there is less repair of the junction between the nerve and the muscle.

“If we block the hyperactive cells responsible for the damage,” he added, “we will increase repair of the junction between the nerve and the muscle, and consequently maintain greater strength, with less muscle destruction, less muscle atrophy and less weakness over time.”

This study follows on from preclinical work carried out by professor Richard Robitaille at the Université de Montréal, in which the treatment improved the strength and mobility of the mice to which it was administered.

“These mice also showed prolonged survival and increased muscle mass,” said Blanchard. “This is new in the sense that the therapies that have been developed in the past have always targeted motor neurons. Nothing had really been looked at for the more peripheral target, which is the junction between the nerve and the muscle.”

Finding a way to repair, or at least slow the degradation of, the neuromuscular junction in patients would represent a major breakthrough, as only two new treatments for ALS - the Lou Gehrig’s disease that was the subject of the famous “ice bucket challenge” several years ago - have been approved in the last thirty years to slow the progression of the disease.

“If the drug is effective in slowing down the disease, that would already be a very big step,” said Blanchard.

However, he warned that patience would be required, as it is more difficult to detect changes in humans than in mice, “since the model is larger”.

What’s more, this study is taking place in a context where the disease has already progressed considerably, whereas it is possible to induce it in mice and intervene at an early stage.

“Maybe we’re not early enough in the disease to see a difference,” said Blanchard. “I think we have a 50-50 chance of being able to demonstrate efficacy.”

If positive signals are ever detected, perhaps it will be possible to demonstrate efficacy in a later phase, with a larger sample size, he concluded. The ultimate goal, of course, would be to have a treatment that could be offered to patients as soon as they are diagnosed.

The researchers are currently recruiting participants for this study.

This report by The Canadian Press was first published in French on Jan. 16, 2025.