Margarida Maia, PhD, science writer —

Stem cells engineered to mature into nerve-supporting cells that produce GDNF — a key factor for motor neuron survival — were safely delivered into the spinal cord of people with amyotrophic lateral sclerosis (ALS), according to data from a Phase 1/2a clinical trial. These cells, dubbed CNS10-NPC-GDNF, were…

People with amyotrophic lateral sclerosis (ALS) have significantly higher blood levels of lead, a toxic heavy metal, than those without the disease, a review study found. While the findings strengthen accumulating evidence of a link between lead exposure and the risk of developing ALS, they do not definitively prove…

Pasithea Therapeutics has gained access to AU$1 million (about $694,000) to further its research into anti-integrin antibodies as potential treatments for amyotrophic lateral sclerosis (ALS). The non-dilutive funds became available following the company’s acquisition of Alpha-5 Integrin, the preclinical biotech company that originally developed the experimental antibodies for…

PandaOmics, an artificial intelligence (AI) platform that looks at vast amounts of data to find “druggable” molecules, has identified more than two dozen new targets with therapeutic potential for amyotrophic lateral sclerosis (ALS), according to a recent study. Target discovery is a first step in the path to…

Corestem is still enrolling participants in its Phase 3 clinical trial of NeuroNata-R (lenzumestrocel), a stem cell therapy that is conditionally approved to treat amyotrophic lateral sclerosis (ALS) in South Korea. The trial, ALSummit (NCT04745299), is evaluating the safety of repeat NeuroNata-R injections and how well…

The U.S. Food and Drug Administration (FDA) has published on its website a five-year action plan meant to help advance the development of — and access to — treatments that may make life better and longer for people with amyotrophic lateral sclerosis (ALS) or other rare neurodegenerative diseases. The…

NeuroSense Therapeutics’ combination therapy PrimeC led to a significant decline in certain biomarkers of amyotrophic lateral sclerosis (ALS) when used as an add-on to standard of care treatment, a study found. Standard treatment alone, meanwhile, appeared to have no impact on these disease-related biomarkers. The preliminary results come from…

A Phase 2b clinical trial evaluating NeuroSense Therapeutics’ experimental combination therapy PrimeC in people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. PARADIGM (NCT05357950) is the company’s next step following the completion of a Phase 2a trial (NCT04165850) wherein the therapy effectively slowed disease…

The Barrow Neurological Institute in Phoenix, Arizona, is calling on people with amyotrophic lateral sclerosis (ALS) and their family members and friends to participate in a biomarker study that aims to create a bank of biological samples and clinical data collected over time. Data will be made available…

One-time delivery of a CRISPR-based gene therapy significantly reduced the buildup of toxic RNA molecules resulting from C9ORF72 mutations in cells and mouse models of amyotrophic lateral sclerosis (ALS), the therapy’s developer, Locanabio, reported. The approach was designed using the company’s CORRECTx platform, which uses CRISPR technology to…