A treatment that could potentially slow the progression of amyotrophic lateral sclerosis 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 therefore retain more strength, and there will be less destruction of the muscle, less damage. muscle atrophy and less weakness over time.
This study stems from preclinical work carried out by Professor Richard Robitaille at the University of Montreal, during which the treatment improved the strength and mobility of the mice to which it was administered. We also observed in these mice a prolongation of survival and an increase in muscle mass, underlined Dr. Blanchard. “It’s new in the sense that therapies that were developed in the past 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 breakdown, of patients’ neuromuscular junction would represent a major breakthrough, since there are 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 over the past 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, you will have to be patient, he warns, because it is indeed more difficult to detect changes in humans than in mice, “since the model is larger”.
Furthermore, this study is taking place in a context where the disease has already progressed significantly, whereas it can be induced in mice and intervened from the start. “Maybe we’re 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 effectiveness.” If positive signals are ever detected, perhaps it will be possible to demonstrate effectiveness in a later phase, with larger sampling, he concluded. The ultimate goal, obviously, would be to have a treatment that could be offered to patients from the moment of diagnosis. Researchers are currently continuing to recruit participants for this study.
Caption and photo credit: Finding a way to repair, or at least slow the breakdown, of patients’ neuromuscular junction would represent a major breakthrough, since there are only two new treatments for ALS – the Lou Gehrig’s disease that made the the subject of the famous “ice bucket challenge” several years ago – have been approved over the last thirty years to slow the progression of the disease. (AP Photo/Walla Walla Union-Bulletin, Greg Lehman, Archives)
