News

Stealth BioTherapeutics announced the initiation of a Phase 1 trial to assess the safety and tolerability of its investigational oral treatment, SBT-272, in healthy volunteers. SBT-272 is being developed to treat neurodegenerative diseases marked by problems in the workings of mitochondria (a cell’s powerhouse), including amyotrophic lateral sclerosis…

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 first patient has been dosed in a Phase 2 trial testing a nanomedicine called CNM-Au8 as a potentially disease-modifying therapy for people with amyotrophic lateral sclerosis (ALS). This safety and efficacy study, due to finish in April 2021, is also completely enrolled, Clene Nanomedicine, the treatment’s developer, announced…

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…