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New Hope for ALS Sufferers

저자:   업로드:2015-07-23  조회수:

Researchers at the CHUM Research Center and the University of Montreal report the discovery of a previously unknown link between the immune system and the death of motor neurons in Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. The scientists believe their research paves the way to an entirely new approach for finding a drug that can cure or at least slow the progression of such neurodegenerative diseases as ALS, Alzheimer's, Parkinson's, and Huntington's diseases.

The study (“Neurodegeneration in C. elegans models of ALS requires TIR-1/Sarm1 immune pathway activation in neurons”), published in Nature Communications, shows that the immune system in the C. elegans animal model, plays a critical role in the development of ALS. “An imbalance of the immune system can contribute to the destruction of motor neurons and trigger the disease,” said Alex Parker, Ph.D., CHUM researcher and associate professor in the department of neuroscience at the University of Montreal.

Amyotrophic lateral sclerosis is a neuromuscular disease that attacks neurons and the spinal cord. Those affected gradually become paralyzed and typically die less than five years after the onset of symptoms. No effective remedy currently exists for this devastating affliction. Riluzole, the only approved medication, extends the patient's life by a few months.

More than a dozen genes are related to ALS. If a mutation occurs in one of them, the person develops the disease. Scientists introduced a mutated human gene (TDP-43 or FUS) into C. elegans. The worms became paralyzed within about 10 days. The challenge was to find a way of saving them from certain death. “We had the idea of modifying another gene (tir-1) known for its role in the immune system,” said Julie Veriepe, lead investigator and doctoral student under the supervision of Dr. Parker. Results were remarkable, noted Veriepe. “Worms with an immune deficit resulting from the tir-1 gene's mutation were in better health and suffered far less paralysis,” she added.

This study highlights a never previously suspected mechanism: even if the C. elegans worm has a rudimentary immune system, that system triggers a misguided attack against the worm's 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,” Dr. Parker explained. Is the same scenario at work with people? Most likely believes Dr. Parker.

The human equivalent of the tir-1 gene—SARM1—has proved crucial to the nervous system's integrity. Researchers think the signaling pathway is identical for all genes associated with ALS. This makes the TIR-1 protein (or SARM1 in humans) an excellent therapeutic target for development of a medication. SARM1 is particularly important because it is part of the well-known kinase activation process, which can be blocked by existing drugs.

Dr. Parker's team is already testing drugs that have been previously approved by the FDA 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 amyotrophic lateral sclerosis.

“In our studies with worms, we know the animal

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