Study to look for potential ALS treatment at Montreal Neurological Institute-Hospital

A treatment that could potentially slow the progression of amyotrophic lateral sclerosis (ALS) is being studied at the Montreal Neurological Institute-Hospital.

Dr. Olivier Blanchard and his team want to know if 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 (disease) process that the junction between the nerve and the muscle is very diseased,” said Dr. Blanchard. “And not only is the neuromuscular junction not good, but there is also 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 the repair of the junction between the nerve and the muscle, we will consequently maintain more strength, and there will be less muscle destruction, less muscle atrophy and less weakness over time.

This study stems from preclinical work carried out by Professor Richard Robitaille at l’Université de Montréal (UdeM), during which the treatment improved the strength and mobility of the mice to which it was administered.

The mice were also observed to have an extension of survival and an increase in muscle mass, Dr. Blanchard said.

“This is new in the sense that the therapies that have been developed in the past have always targeted motor neurons,” he said. “Nothing had really been looked at for the more peripheral target, 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 be a major breakthrough, since only two new treatments for ALS – Lou Gehrig’s disease, which 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 the disease, that would already be very big,” said Dr. Blanchard.

However, patience will be required, he warns, because it is more difficult to detect changes in humans than in mice, “since the model is larger.”

In addition, this study is taking place in a context where the disease has already progressed significantly, whereas it can be induced in mice and intervened early on.

“Maybe we are not early enough in the disease to see a difference,” said Dr. Blanchard. “I think we have a one in two 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, he said.

The ultimate goal, he says, would be to have a treatment that could be offered to patients from the moment of diagnosis.

The researchers are currently continuing to recruit participants for this study.

–This report by La Presse Canadienne was translated by CityNews