Lindsey Shapiro, PhD,  science writer—

Treatment with CNM-Au8 was shown to significantly prolong survival — relative to the natural history of the disease — among people with advanced amyotrophic lateral sclerosis (ALS) who took part in two expanded access programs (EAP), also known as compassionate use programs. The findings from these EAPs, which…

Denali Therapeutics’ SAR443820 (DNL788) treatment candidate — now in late-stage clinical testing for amyotrophic lateral sclerosis (ALS), per the company’s website — has failed to significantly slow disease progression in adults in a Phase 2 trial. The experimental therapy “did not meet the primary endpoint of change” in outcomes…

FundaMental Pharma’s investigational molecule FP802 reduced neurological disability, protected nerve cells, and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to recent research. The molecule also protected tissue from glutamate toxicity, a type of cellular toxicity that’s implicated…

Mutations in the C9ORF72 gene may lead to amyotrophic lateral sclerosis (ALS) and the related disorder frontotemporal dementia (FTD) in part by boosting the production of a powerful inflammatory protein called interleukin 17A (IL-17A), recent preclinical research suggests. Inhibiting IL-17A via genetic manipulations or…

On the heels of promising HEALEY-ALS Phase 2 trial data, Prilenia Therapeutics plans to launch a pivotal Phase 3 study of pridopidine, its candidate amyotrophic lateral sclerosis (ALS) treatment, in the second half of this year. Relative to a placebo, pridopidine was associated with slower disease progression,…

Blocking certain inflammatory signaling pathways may be one way to reverse nerve cell alterations that drive the symptoms of amyotrophic lateral sclerosis (ALS), according to preclinical research using a mouse model of the disease. In the mice, an ALS-associated mutation in the FUS gene caused caused motor neurons — the…

Up to three years of daily treatment with the investigational therapy IPL344 was well tolerated and slowed disease progression among adults with amyotrophic lateral sclerosis (ALS), according to top-line data from a small Phase 1/2a clinical trial. Preliminary efficacy analyses also indicated that IPL344 might be able to…

Researchers have developed an algorithm to generate personalized survival predictions for people with amyotrophic lateral sclerosis (ALS) based on clinical and MRI features. The scientists believe the algorithm could help patients and doctors make better treatment and care plans. “The disease progression varies greatly for ALS patients, so it…

The ALS Association has given Oryzon Genomics nearly $500,000 to advance ORY-4001, a potential treatment of amyotrophic lateral sclerosis (ALS), into preclinical studies. The $498,690 grant was provided via the association’s Lawrence and Isabel Barnett Drug Development Program. Oryzon intends for laboratory studies in mouse disease models…

Genetic and clinical factors that can influence how amyotrophic lateral sclerosis (ALS) progresses were identified in a recent analysis of data from PRECISION-ALS, a large European research project. Consistent with known risk factors for ALS progression, C9ORF72 genetic mutations, bulbar-onset disease, and faster functional declines were linked…