The National Institutes of Health (NIH) has awarded about $45.1 million toward an expanded access program (EAP) of CNM-Au8, an oral therapy candidate for amyotrophic lateral sclerosis (ALS). The EAP, commonly referred to as compassionate use, will make CNM-Au8 available to U.S. patients who aren’t eligible for clinical…

An advisory committee of the European Medicines Agency has maintained its recommendation not to approve AMX0035 for amyotrophic lateral sclerosis (ALS). In its initial assessment issued in June, the Committee for Medicinal Products for Human Use (CHMP) said data from the CENTAUR Phase 2 trial (NCT03127514), which formed…

People with amyotrophic lateral sclerosis (ALS) due to mutations in the FUS gene may benefit from interferon-gamma treatment to delay disease onset and/or progression, an early preclinical study suggests. Researchers found that an immune signaling protein called interferon (INF)-gamma protected patient-derived motor neurons from oxidative stress. INF-gamma also restored…

The Barrow Neurological Institute has received $16.7 million from the National Institutes of Health (NIH) to help coordinate a nationwide repository of clinical data of people with amyotrophic lateral sclerosis (ALS). The Access for All in ALS (ALL ALS) Consortium will serve as a repository of biological samples and clinical…

PrimeC, NeuroSense Therapeutics‘ investigational, fixed-dose combination therapy for amyotrophic lateral sclerosis (ALS), significantly boosted the survival of patient-derived motor neurons to levels similar to healthy controls in a lab study. The independent study was led by Justin Ichida, PhD, a professor of stem cell biology and regenerative…

Amyotrophic lateral sclerosis (ALS) patients not eligible to participate in clinical trials of pridopidine — a small molecule developed by Prilenia Therapeutics to slow disease progression in ALS — will soon be able to receive the experimental therapy via an expanded access program (EAP). EAPs, also known…

Cytokinetics is calling for proposals from patient advocacy organizations focused on amyotrophic lateral sclerosis (ALS) and cardiovascular conditions for its 6th annual Communications Grant Program. The 2024 program will award five grants totaling $20,000 each to groups serving the ALS, heart failure, and hypertrophic cardiomyopathy communities. These…

Spinogenix has won a nearly $1 million grant from the U.S. Department of Defense (DoD) to move ahead with clinical testing of SPG302, a small molecule candidate for amyotrophic lateral sclerosis (ALS). This is DoD’s Congressionally Directed Medical Research Programs’ second grant to the company, following a…

Direct measures of muscle strength were correlated with functional abilities among amyotrophic lateral sclerosis (ALS) patients in a Phase 2 clinical trial. Measures of arm and hand muscle strength aligned well with functional domains related to fine motor skills (e.g., handwriting, grasping), whereas leg and foot strength were related…

Takeda has obtained an exclusive, worldwide license to AcuraStem’s investigational therapies targeting the PIKfyve enzyme, including AS-202, a potential treatment for amyotrophic lateral sclerosis (ALS) that ultimately aims to lower toxic TDP-43 protein buildup in nerve cells. While Takeda will be responsible for clinical development,…