Marisa Wexler, MS, senior science writer —

Stem cell therapy, specifically treatment with mesenchymal stem cells or MSCs, did not substantially alter disease progression among people with amyotrophic lateral sclerosis (ALS), according to new data from a Phase 2 clinical trial. However, there was a subset of patients who did appear to experience notable benefits from…

In people who experience a traumatic brain injury, or TBI — which is linked to a greater likelihood of developing amyotrophic lateral sclerosis (ALS) — blocking the activity of a protein called KCNJ2 may lower the risk of ALS, according to new research done in lab models. “Targeting KCNJ2…

Taking part in certain recreational activities, including gardening, golfing, woodworking, and hunting, may increase the risk of developing amyotrophic lateral sclerosis (ALS), especially for men, according to a new study. While the causes of ALS still are not well understood, several risk factors have been identified. There’s evidence…

Blocking a protein called PTP sigma led to better nerve survival and motor function in a mouse model of amyotrophic lateral sclerosis (ALS) caused by mutations in the C9ORF72 gene — leading the researchers to highlight this strategy for potentially treating this genetic ALS form. Data from cell models…

Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for people with amyotrophic lateral sclerosis (ALS), has failed to meet its primary goal of slowing disease progression in an ongoing Phase 3 clinical trial designed to confirm its effectiveness. That’s according to new data from the trial, dubbed PHOENIX,…

New data show that blood-based measures, including levels of markers of nerve damage and chemical DNA modifications, can help predict which individuals with amyotrophic lateral sclerosis (ALS) will have faster disease progression over time. However, because the disease course may be altered with Radicava or Radicava ORS —…

Note: This story was updated March 6, 2024, to correct the poster was presented by Brainstorm’s executive vice president and chief development officer Bob Dagher. A pivotal, Phase 3b clinical trial is planned to test the cell-based therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS),…

While the most recent guidelines for treating amyotrophic lateral sclerosis (ALS) recommend that patients receive three available treatments together, as soon as possible, a new analysis of U.S. data shows that most individuals with the progressive disease don’t get this combination in clinical practice. The analysis found that most…

Health Canada has issued a notice of noncompliance-withdrawal saying it won’t approve the oral therapy masitinib as an add-on treatment for amyotrophic lateral sclerosis (ALS). Its developer, AB Science, can submit a request for reconsideration within the next month, which it plans to do, it announced in…

An experimental gene therapy called CTx1000, which is designed to reduce toxic buildups of the protein TDP-43 in nerve cells, was found to significantly extend survival and improve motor function in mouse models of amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia, known as FTD. According to…