New Treatment Hope for Amytrophic Lateral Sclerosis

In a recent discovery by scientists at the CHUM Research Centre and the University of Montreal, a previously unknown link was found between the immune system and the death of motor neurons in amytrophic lateral sclerosis (ALS). The findings could potentially offer a new approach for developing drug treatments that can slow the progression of neurodegenerative diseases such as ALS, Alzheimer’s disease, Parkinson’s disease and Huntington’s disease. The study, published in the journal Nature Communications, shows that the immune system in the animal model C. elegans, a tiny one millimeter-long roundworm, plays a critical role in the development of ALS. More than a dozen genes are related to ALS, and if a mutation occurs in one of them, the person will develop the disease. Scientists introduced a mutated human gene (TDP-43 or FUS) into C. elegans, a nematode worm widely used for genetic experiments. The worms became paralyzed within about 10 days, and the research team’s challenge was to find a way of saving them from dying. This study highlights a never previously suspected mechanism — even if the C. elegans worm has a very rudimentary immune system, that system triggers a misguided attack against the worms’ own neurons. “The worm thinks it has a viral or bacterial infection and launches an immune response. But the reaction is toxic and destroys the animal’s motor neurons,” said a lead researcher of the study. The research team is actively testing drugs that have been previously approved by the U.S. Food and Drug Administration for treatment of such disorders as rheumatoid arthritis, to see if they work with ALS. Obstacles still remain, however, before finding a remedy for curing or slowing the progression of the disease. To read more about this study, click here.