Toxic clumps due to the buildup of the TDP-43 protein can trigger the neuronal inflammation that precedes symptoms of amyotrophic lateral sclerosis (ALS) by activating an inflammatory signaling pathway called cGAS-STING, a study reported. Its findings suggest that targeting this inflammatory signaling may be an effective way of treating ALS.
News
A Phase 1/2 clinical trial investigating ION541, Ionis Pharmaceuticals‘ third antisense treatment and its first for sporadic, rather than familial, amyotrophic lateral sclerosis (ALS), has started dosing, the company announced. This ascending dose safety trial (NCT04494256) is recruiting up to 70 patients…
People with diabetes mellitus, particularly those with type 2 diabetes, appear to be at a significantly lower risk of amyotrophic lateral sclerosis (ALS) than those without this metabolic disorder, a review study reported. Its findings support previous research suggesting diabetes to be a protective factor against ALS, potentially…
Catalent Biologics has agreed to manufacture NurOwn, the cell-based therapy by BrainStorm Cell Therapeutics being evaluated in a soon-to-conclude pivotal trial as a possible treatment of amyotrophic lateral sclerosis (ALS). With this agreement, Catalent will produce NurOwn under current Good Manufacturing Practices — standards set to ensure that batches…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
MND-SMART, a pivotal platform trial designed to simultaneously test multiple treatment candidates in people with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases, has opened its third clinical site in Scotland. This type of platform trial, distinct from typical clinical trials that test a single treatment…
A $2 million grant from the National Institutes of Health (NIH) is funding work to better understand how molecular structures called protein-RNA condensates form and are regulated within cells. Findings from the five-year project could help in understanding and treating diseases where these molecular structures play a role, including…
Head trauma, electrical burns, and hobbies involving the toxic metal lead appear to be risk factors for developing amyotrophic lateral sclerosis (ALS), a U.S.-based study found. Holding a job in mechanics, painting, or construction also significantly increase the chances of developing the disease, researchers noted. The study, “…
A metabolite of testosterone, dihydrotestosterone (DHT), is found at significantly lower levels in the cerebrospinal fluid of people with amyotrophic lateral sclerosis (ALS), a small study found. This metabolite, or hormone, is likely critical for the survival of motor neurons, and its lack in the central…
Treatment with AMX0035, Amylyx’s experimental oral therapy, significantly extends the lives of amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to a long-term survival analysis of the CENTAUR Phase 2/3 trial. The new data were reported in a study titled “Long‐Term…
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