'als will be a treatable chronic disease': new treatment slows disease progression and increases life expectancy

eligible canadians who currently live with amyotrophic lateral sclerosis (als) – a devastating progressive neurodegenerative disease — may soon be able to access a promising new treatment. a clinical trial with 137 participants, conducted over a 24-week period, demonstrates that albrioza™ , also known as amx0035, could help slow down the progression of the disease.

the investigative treatment that’s raising hope for the als community across the country was recently approved by health canada with conditions. joshua cohen and justin klee conceived the treatment back in 2013 while attending brown university as a way to help prevent the loss of neurons that typically occur in brain disorders. since then, they’ve founded amylyx pharmaceuticals and have taken amx0035 through clinical trials and regulatory action.

amx0035 is intended to reduce nerve cell death by obstructing cellular death pathways in the mitochondria and endoplasmic reticulum . often referred to as the “ powerhouse of the cell ,” mitochondria provide the energy for cell’s survival while the endoplasmic reticulum is responsible for producing proteins that are necessary for cell functioning. if either one of these important compartments fails to properly carry out its function, it could result in cell death or inflammation , both of which may contribute to the development of als.

when used either as a stand-alone intervention or in conjunction with an existing treatment, amx0035 appears to have meaningful benefits on function as well as survival for those with als, based on results of the phase 2 clinical trial ( centaur ). the therapy which uses a combination of two key compounds — sodium phenylbutyrate and taurursodiol – has shown that it could help maintain muscle strength and function longer when compared to the placebo group. in fact, trial participants who received the treatment scored 2.32 points higher on the amyotrophic lateral sclerosis functional rating scale – revised (alsfrs-r) over a period of 24 weeks — alsfrs-r is a tool that is used to determine the severity of the disease at the time of diagnosis and assess its progression over time.

dr. angela genge, executive director of the clinical research unit at the montreal neurological institute at mcgill university spoke with healthing about amx0035, what it means for patients, and why she believes that als could become a treatable chronic disease in the future.

why is amx0035 so significant in the fight against als?

what is its success rate?

when do you expect canadian patients to have access?

as someone who treats patients with als, what are you most excited about?

what made you get into neurology?

  the last frontier in terms of therapies that work effectively. when i began my career, multiple sclerosis (ms) was not treatable, there were no therapies for dementia, or als. now, there are 15 treatments for ms and early disease modifying therapies for both alzheimer’s and als. the future for both looks good.