'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 approvedby 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 pharmaceuticalsand have taken amx0035 through clinical trials and regulatory action.

amx0035 is intended to reduce nerve cell deathby obstructing cellular death pathways in the mitochondriaand 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 functionas well as survivalfor 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.

full health canada approval is expected once the company provides data from the ongoing third phase of the clinical trial and makes top line results of the current clinical trial available which are anticipated in 2024. the approval with conditions makes amx0035 only the third als treatment option available to canadians, and eligible patients could have access to it as early as next month.

dr. angela genge, executive director of the clinical research unit at the montreal neurological institute at mcgill universityspoke 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?

amx0035 has been shown to slow down the loss of function. in other words, strength and therefore function is maintained for longer. as a consequence of this, quality of life can be better as abilities are better for longer.

what is its success rate?

rather than success rate, the better measure is the difference in function at the end of six months and the life expectancy. both are significantly better on amx0035.

when do you expect canadian patients to have access?

in canada, health canada approval is just the first step. some insurance companies will approve it right away, however, the general public must wait for a process involving quebec and national committees to review the file and agree to provide it in each of the 10 provinces.

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

treatments for this terrible disease will come, science is moving us forward all the time. every step gives our patients more time, better function for longer, better care every year. we will overcome this disease and als will become a treatable chronic disease.

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.