News

AMX0035 — Amylyx Pharmaceuticals’ experimental combination therapy for amyotrophic lateral sclerosis (ALS) now under review for approval in the U.S. — alters the activity of genes involved in several disease-relevant pathways, according to a study in lab-grown cells. Notably, the two-compound oral treatment was seen to change the activity of many…

The levels of five small RNA molecules, called microRNAs, inside tiny vesicles in the blood of people with amyotrophic lateral sclerosis (ALS) differ significantly from those of people without ALS and may offer a way of accurately and quickly diagnosing the disease, a study suggests. Importantly, its researchers evaluated…

A treatment known as VPA-Li, a combination of lithium carbonate and valproic acid, may slow disease progression and improve quality of life for people with amyotrophic lateral sclerosis (ALS), according to data from a small Phase 2 clinical trial. Favorable trends in survival and lung health were also observed…

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…

In a reversal, an advisory committee of the U.S. Food and Drug Administration (FDA) now says that the current evidence of efficacy of Amylyx Pharmaceuticals’ AMX0035 is sufficient to support its approval for amyotrophic lateral sclerosis (ALS). The committee voted 7–2 in favor of AMX0035, Amylyx announced…

Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…

Higher blood levels of antibodies against the viral protein HERV-K ENV are associated with a better survival rate and improved outcomes in people with amyotrophic lateral sclerosis (ALS), a new study shows. These findings further support the therapeutic potential of GNK301, an experimental antibody against HERV-K ENV being…

To help expand research into the treatment and cure of amyotrophic lateral sclerosis (ALS), the Blazeman Foundation for ALS has donated $800,000 to the University of Maryland School of Medicine (UMSOM). The funds will be used to boost research at the University of Maryland Brain and Tissue Bank,…

Treating patients with their own immune regulatory T-cells (Tregs), in combination with IL-2 — a protein that boosts the cells’ immunosuppressive function and survival — is generally safe and shows potential to slow disease progression in people with amyotrophic lateral sclerosis (ALS). That’s according to a small Phase…

The cerebrospinal fluid of people with sporadic amyotrophic lateral sclerosis (sALS), but not of those with familial forms of the disease, promoted ALS-specific molecular, cellular, and motor abnormalities in healthy mice, a study showed. Apolipoprotein B-100 (apoB100), a protein involved in cholesterol transport, was identified as the main…