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Treatment with Ultomiris (ravulizumab-cwvz) — a therapy approved for several rare diseases that works by blocking activation of part of the immune system called the complement cascade — failed to slow the progression of amyotrophic lateral sclerosis (ALS) in a Phase 3 clinical trial that had been launched…

Inhibiting the NLRP3 inflammasome — an immune system protein complex believed to contribute to the inflammation that drives amyotrophic lateral sclerosis (ALS) — alone was not sufficient to ease spinal cord inflammation in a mouse model of the disease, according to recent research. NLRP3 is overactive in ALS and…

Bryostatin-1, a molecule being developed to treat amyotrophic lateral sclerosis (ALS), improved cell survival in a lab model of the disease, a new study reports. The therapy is known to activate PKC-epsilon, a protein that’s significantly reduced in ALS patients’ motor cortex, a brain region involved in muscle movement…

Aruna Bio‘s investigational therapy AB126 significantly eased inflammation in the spinal cord and extended survival in a mouse model of amyotrophic lateral sclerosis (ALS), the company announced. The therapy, which is expected to treat several other neurodegenerative and neuroinflammatory disorders, also significantly reduced the levels of neurofilament…

Having a family member with amyotrophic lateral sclerosis (ALS) can strain household finances as well as the emotions of caregivers and other relatives. To help relieve some of that financial burden, the Jane Calmes ALS Scholarship Fund was established four years ago to support post-high school education for…

A newly developed cell-based system, made of nerve cells connected to muscle fibers, may allow the rapid, large-scale screening of potential medicines for neuromuscular conditions, such as amyotrophic lateral sclerosis (ALS). Applied automated imaging methods, which are responsive to the new system, can be used to visualize and measure…

Treatment with regulatory T-cells (Tregs), a type of anti-inflammatory immune cell, reduced biomarker levels of inflammation and oxidative stress in people with amyotrophic lateral sclerosis (ALS), a study shows. The experimental approach may help curb these contributions to nerve cell damage in ALS, the findings suggest, and routinely…

When the TDP-43 protein is less able to form two-protein complexes called dimers, it appears to be more likely to form the toxic clumps seen in amyotrophic lateral sclerosis (ALS), a study reports. As these clumps are found in about 97% of all ALS cases, a better understanding of the…

Note: This story was updated Sept. 25, 2023, to correct that TW001 uses the same active ingredient as Radicava and Radicava ORS but is a different therapy. Treeway has chosen Specialised Therapeutics Asia, known as ST, as its partner to market TW001, an oral formulation of edaravone, for…

Mutations in the C9ORF7 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), lead to defects in the stem cells needed for brain development in the early stages of life, a new study shows. In animal models of the disease, this resulted in smaller than normal volumes…