als treatment breakthrough 30 years in the making
4 minute read
the goal of dr. michael strong and the team at western university is to translate the results of their innovative als research into human trials in the next five years.
allan lewis, schulich school of medicine & dentistry
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a breakthrough in the study of amyotrophic lateral sclerosis (als) has found a potential path towards finding a cure for the disease.
the results of the study, conducted by dr. michael strong and a team of western university researchers, were published in the journal brain and highlight how specific interactions with proteins can help prevent the death of nerve cells in those with als.
it was no easy feat to arrive at these results, as this research is the sum of roughly three decades of work surrounding als conducted by dr. strong and associates, and backed by the temerty foundation, a non-profit dedicated to improving the health and lives of those living in canada through research and innovation in the healthcare space.
als and the research
als is a motor neuron disease that develops when nerve cells in the brain and spinal cord become impaired, leading to symptoms such as paralysis, muscle wastage, and, eventually, death. current treatments in the als space aim to slow the progression of the disease while alleviating symptoms, but so far, none have been effectively coined as a cure.
dr. strong, who is both a medical researcher and clinician who works with als patients and their families, has dedicated his life to finding a cure for als. that commitment has driven the 30 years of research to arrive at this point, where the path toward a cure may now be clearing. the discovery is centred around a protein and its interaction with nerve cells.
the protein in question—tdp-43—is the culprit behind als, acting as the instigator for the development of the disease by forming abnormal clumps within nerve cells, causing them to die. while this protein is well-established as part of the reason for the onset of als, a second protein discovered by the team has the power to do the opposite.
sponsored by:
als society of canada (als canada)
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the rgnef protein has a specific fragment, nf242, that works to stop or discourage the toxicity of tdp-43. essentially, this small fragment can change the entire game for those living with als because of how the two proteins interact.
when tdp-43 and the nf242 come into contact with one another, nf242 has the ability to counteract the mechanism of action of the tdp-43 protein, essentially stopping the damage being done to the neurons.
the study was conducted using fruit flies with an average lifespan of 60 to 90 days. within two weeks, the fruit flies in the study that had als were unable to walk. however, when the protein fragment was introduced into the flies, function was restored, and the flies lived out their regular 60- to 90-day lifespan because it mitigated the effects of the damaging tdp-43 protein.
another version of the study was done in mice, and the results were similar, showing extra promise in the viability of using nf242 for treating, or potentially curing, als.
changing the course of als and other neurological conditions
people who develop als have a significantly reduced quality of life and life expectancy. typically, the life expectancy after being diagnosed with als is roughly two to five years.
however, if the results indicated in the animal-based study translate into humans, it could essentially change the course of als in people in a way that no other medication or medical intervention has been able to yet.
the tdp-43 protein is also associated with other debilitating neurological conditions, including:
- alzheimer’s disease
- chronic traumatic encephalopathy
- lewy body disease
- huntington’s disease
- argyrophilic grain disease
- hippocampal sclerosis
- frontotemporal dementia
because of the way the protein reacts in these diseases, and als, and the counteraction of nf242, not only can this discovery pave the way for an als cure, but also potential treatments in all other diseases that are affected by tdp-43 protein buildup.
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the next steps
the research, while showing promise, still has to be tested in those living with als and other neurodegenerative disorders for it to be considered a viable path toward treating these diseases in a way that provides hope to those living with als and their families.
the goal of dr. strong and the team at western university is to translate the results into human trials in the next five years. while that may seem like a long time, after working on this for decades, dr. strong and the team can finally see a way forward when it comes to using these proteins to benefit als patients.
“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,’” strong said in a release from western university. “it’s been 30 years of work to get here; 30 years of looking after families and patients and their loved ones, when all we had was hope. this gives us reason to believe we’ve discovered a path to treatment.”
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