Canadian researchers unlock potential ‘cure’ for ALS

By Ashley Hyshka

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    LONDON, ONTARIO (CTV Network) — New research out of London, Ont.’s Western University is shedding light on a potential cure for ALS, in which the targeting of the interaction between two proteins can halt or fully reverse the disease’s progression.

According to a news release from Western University on Monday, a team of researchers, led by Dr. Michael Strong, have discovered a potential path toward a cure for amyotrophic lateral sclerosis, also known as ALS.

“As a doctor, it’s been so important for me to be able to sit down with a patient or their family and say to them, ‘We’re trying to stop this disease,’” said Strong, Arthur J Hudson Chair in ALS research at Western’s Schulich School of Medicine & Dentistry.

Published in the journal Brain, Strong’s team discovered that targeting an interaction between two proteins present in ALS-impacted nerve cells can halt or even fully reverse the progression of the disease.

“Importantly, this interaction could be key to unlocking a treatment not just for ALS but also for other related neurological conditions, like frontotemporal dementia,” said Strong. “It is a game changer.”

According to the study, in nearly all ALS patients, a protein called TDP-43 is responsible for forming abnormal clumps within cells, which causes cell death. In recent years, Strong’s team discovered a second protein, called RGNEF, with functions that are opposite to TDP-43.

The team’s research identified a specific fragment of the RGNEF protein — named NF242 — that can “mitigate the toxic effects of the ALS-causing protein.”

As a result, the researchers discovered that when the two proteins interact with each other, the toxicity of the ALS-causing protein is removed, which significantly reduces damage to the nerve cell and therefore prevents its death.

After 30 years of his life working on ALS research with various teams, Strong is confident in what the future holds as a result of the new discovery not just for the treatment of ALS, but for other neurodegenerative diseases.

“I think we can reasonably look at this and say, ‘This is swinging for the bleachers,’ we think in three to five years, we might be looking at something [ALS] that might be cured,” said Strong. “We can, in an experimental model, significantly either completely abolish the [ALS] disease process. Or, in another one do a significant job of slowing it down and changing the course of the disease.”

How researchers got here

The light bulb moment in Strong’s research came seven years ago when the team was working with fruit flies. Flies that were genetically modified to have the pathology of ALS died within 10 to 14 days, whereas a fly typically has a lifecycle of 70 to 80 days.

Those flies with the new pathway Strong said had a longer lifespan, improved motor functions and their nerve cells were protected from degeneration.

The next step in research was using mice, where a mouse named Lucy who had been genetically modified to have ALS was given both treatments.

Her sister did not receive the treatment and died, whereas Lucy lived through experimentation and according to Strong was “doing fine.”

“Best results I’ve ever seen from an animal model,” he said.

What is ALS?

Also known as Lou Gehrig’s disease, ALS is a debilitating neurodegenerative condition that progressively impairs nerve cells responsible for muscle control, leading to muscle wastage, paralysis and death, according to Western.

The average life expectancy of an ALS patient post-diagnosis is two to five years.

Due to the complex nature of the disease, there are no effective forms of therapy that can prevent its progression.

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