News

Mitochondrial abnormalities seen early in many forms of ALS: Study

A study found that genetic mutations associated with amyotrophic lateral sclerosis (ALS) lead to problems with the function and transport of mitochondria, cellular structures needed for energy production, early in the disease course, before abnormal protein clumps are formed. “We show that the nerve cells, termed motor neurons, that…

ALS Network collaborates on $300K drug-repurposing study

The ALS Network will support a collaborative research effort, at California’s Lawrence Livermore National Laboratory, that will use electronic health records (EHRs) to identify existing medications that could slow the progression of amyotrophic lateral sclerosis (ALS). The $300,000 project is aimed at drug repurposing — finding medications already…

FDA clears Phase 3b trial of NurOwn for ALS

The U.S. Food and Drug Administration (FDA) has cleared a Phase 3b clinical trial to test Brainstorm Cell Therapeutics’ cell-based therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS). The trial, ENDURANCE (NCT06973629), is expected to open soon at more than a dozen sites across…

ATH-1105 experimental therapy found safe in healthy adults: Trial

ATH-1105, an experimental oral therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, was found to be safe and well tolerated in healthy volunteers, according to the treatment’s developer, Athira Pharma. The data comes from a first-in-human Phase 1 trial (NCT06432647) that tested the safety and pharmacological…

Adding low-dose IL-2 to riluzole may improve survival in ALS

Adding a low dose of the anti-inflammatory molecule interleukin-2 (IL-2) to treatment with riluzole is safe and may help extend survival in certain people with amyotrophic lateral sclerosis (ALS), according to findings from a clinical trial called MIROCALS. The Phase 2b trial didn’t show a significant survival benefit in…

Neuronata-R improved function, survival in slow-progressing ALS

Neuronata-R (lenzumestrocel), an investigational stem cell therapy from Corestemchemon significantly improved function and reduced markers of nerve cell damage in a subgroup of people with slow-progressing amyotrophic lateral sclerosis (ALS), the final results of a Phase 3 clinical trial show. The ALSummit trial (NCT04745299) failed to meet…

Young investigators receive MGH awards for ALS research

Three researchers at Massachusetts General Hospital (MGH) have received 2025 MGH ALS Young Investigator Awards for their research on genetic abnormalities, faulty energy systems, and why some nerve cells are more susceptible to damage than others in amyotrophic lateral sclerosis (ALS). The awards announced by the Sean M.