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…
Dosing of healthy volunteers has been completed in a Phase 1 clinical trial evaluating Neos Therapeutics‘ experimental therapy NT0502 for chronic sialorrhea (excessive drooling), a common problem in people with amyotrophic lateral sclerosis (ALS). The open-label study includes 30 healthy adults who were assigned randomly to take one of…
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…
Amyotrophic lateral sclerosis (ALS) patients receiving the lowest dose of Kadimastem’s cell therapy candidate, AstroRx, experienced a significant reduction in disease progression in the three or four months after treatment, updated findings from the company’s Phase 1/2 clinical trial show. In subsequent…
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…
