The U.S. Food and Drug Administration (FDA) has authorized the evaluation of the first three candidate therapies for amyotrophic lateral sclerosis (ALS) in the HEALEY ALS Platform Trial. This trial model, designed to accelerate the development of ALS therapies by assessing multiple treatment candidates simultaneously, is the first of…

Athletes who played professional football — known as soccer in the U.S. — are more than three times more likely to die of a neurodegenerative disease than are the general population, a study from Scotland has found. Its researchers compared causes of death over 18 years for about 7,700 former professional…

A single spinal injection of an investigational RNA therapy blocked motor neuron degeneration and saved motor function in animal models of familial amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations, a study  reports. Giving the treatment — a virus-delivered gene-silencer targeting the SOD1 gene —  before the onset of…

A recently discovered inflammatory mechanism triggered by Schwann cells —  specialized cells that produce the protective myelin sheath around neurons —  in amyotrophic lateral sclerosis (ALS) can be alleviated with masitinib, a study in rat models shows. The study, “Schwann cells orchestrate peripheral nerve…

To help accelerate development of effective treatments for amyotrophic lateral sclerosis (ALS), the ALS Association is investing $3 million in the disease’s first platform trial. The award is $1 million annually for three years to support the platform investigation, a type of clinical study that evaluates the…

A recently completed Phase 1 trial studying NPT520-34 — an investigational small molecule being developed by Neuropore Therapies for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS) — has shown the candidate to be safe and well-tolerated in a group of healthy volunteers. The…

In the coming months, Alexion Pharmaceuticals will launch a pivotal Phase 3 clinical trial to test its complement system inhibitor Ultomiris (ravulizumab) in people with amyotrophic lateral sclerosis (ALS) whose motor symptoms began in the prior three years. The CHAMPION-ALS trial — which follows an investigational new…

Including ALS Research Ambassadors — a group of amyotrophic lateral sclerosis (ALS) patients and their caregivers — in discussions about future clinical trial planning and design facilitates changes in study protocol that make the studies more patient-centered. That result is shown in the case of the REFINE-ALS trial, which is…

Kadimastem has finished treating its second group of participants in a Phase 1/2a clinical trial testing the safety and preliminary efficacy of AstroRx, an investigational stem cell therapy for amyotrophic lateral sclerosis (ALS). AstroRx is an off-the-shelf cell therapy consisting…

A newly discovered self-destructive mechanism in mitochondria, the cells’  powerhouses, may be one of the first deficits leading to motor neuron degeneration associated with toxic TDP-43 clumps — a hallmark of amyotrophic lateral sclerosis (ALS). That evidence from a preclinical study suggests available therapies against mitochondrial degeneration might help halt neurodegeneration…