Top 10 ALS Stories of 2022
New treatments and approvals top the list of key ALS stories
We look forward to continuing to serve as a resource for the ALS community in the new year. Here, we’ve compiled a list of the top 10 most-read stories we published this past year.
Amylyx Pharmaceuticals developed an oral therapy, called AMX0035, containing a fixed combination of two molecules thought to help protect nerve cells from damage and stress. In June, the therapy was granted conditional approval by Health Canada under the brand name Albrioza. This marked the first new ALS therapy to be approved in Canada since 2018, and the first ever regulatory approval for the investigational therapy.
In February, Amylyx announced it would be holding a meeting with an advisory committee of the U.S. Food and Drug Administration (FDA) to discuss potential approval of AMX0035 in for the treatment of ALS. While the committee first voted narrowly that there was not enough data to support therapy’s efficacy, it would vote in favor of the therapy in September, in light of new data.
About a month after Albrioza’s conditional approval by Health Canada, Amylyx announced that it was ready to fill prescriptions for the medication in the country. The company said it would work with decision-makers and insurers in the country to make the therapy available to patients. Amylyx also offers patients help with navigating insurance, coordinating treatment, and educational support through its Amylyx Care Team Support Program.
In late September, Amylyx announced that the FDA had approved AMX0035 for adults with ALS under the brand name Relyvrio. It also said that the therapy would be marketed in the U.S. with a list price of approximately $158,000 per year. The approval was welcomed by patient organizations. The Amylyx Care Team Support Program is also available for U.S. patients.
The U.S. and Canada approvals of AMX0035 were supported by data from the Phase 2 CENTAUR clinical trial (NCT03127514). After six months in the initial placebo-controlled study, participants had the option to enroll in an open-label extension study (NCT03488524), where all were treated with the then-investigational therapy. Scientists at Amylyx and other institutions conducted statistical tests to assess the effect of treatment with AMX0035 on survival outcomes, compared to what would be expected if patients on placebo had continued to be given placebo rather than switching to active treatment in the extension study. Results indicated that treatment extended median survival time by 10.6 months, which translated to a 61% lower risk of death.
Radicava (edaravone) is an approved ALS therapy sold by Mitsubishi Tanabe Pharma America (MTPA) administered via infusions into the bloodstream. MTPA developed an oral version of the therapy aiming to make treatment more convenient for patients. The company announced in May the FDA had approved the new oral formulation, based on pharmacological and clinical data suggesting that it has a similar efficacy profile and is generally well-tolerated.
The gut microbiome — the billions of bacteria and other microorganisms that live in the digestive tract — has a profound effect on health and disease that is only beginning to be understood. Researchers conducted a study in a mouse model of ALS caused by mutations in the SOD1 gene and found that these mice had abnormalities in digestive function and alterations in gut microbiome composition. Notably, these changes were evident even before the mice started to develop any signs of ALS-like disease. The alterations resolved when mice were given an anti-inflammatory molecule produced by certain gut bacteria, suggesting that therapies targeting the gut microbiome could be beneficial to ALS patients with SOD1 mutations.
In the most advanced “locked in” stage of ALS, patients are completely unable to move voluntarily, although they are fully aware of their surroundings. Researchers described the case of a 34-year-old man whose ALS had progressed to the point that he could no longer reliably control his eye movements, so he couldn’t use eye-tracking technologies to communicate. An electrode was implanted into the man’s brain, and he learned to control his thought patterns to modulate a tone up or down. Using this system, he was able to communicate with others around him, making requests for his care and interacting with his family and loved ones.
Vitamin B12 is important for maintaining nerve health, and early trials suggested that ultrahigh doses of methylcobalamin (the active form of B12) may slow ALS progression in patients with moderately progressing disease. The Phase 3 JETALS trial (NCT03548311) enrolled 130 people with early, moderately progressing ALS, who were given high-dose methylcobalamin or placebo injections for about four months. The trial met its main goal: the rate of disease progression was more than 40% slower in methylcobalamin-treated patients, and high doses of the vitamin were generally well-tolerated.
Cerebrospinal fluid, or CSF, is the liquid that surrounds the brain and spinal cord. Here, researchers found that CSF taken from people with sporadic ALS, but not familial forms of the disease, induced ALS-like symptoms in mice. Further analysis revealed this effect was mainly driven by the activity of a cholesterol transport protein called ApoB100 — injecting this protein alone into mice was sufficient to cause ALS-like disease, while removing the protein prevented disease from developing. Researchers proposed that this protein may be a useful therapeutic target in ALS.
We at ALS News Today hope these stories and all of our reporting in 2022 have helped to inform and improve the lives of people in the ALS community, and we look forward to continuing to be a service for the community in 2023.
We wish all our readers a happy 2023!