The TREM2 stabilizer failed to show efficacy in a Phase 2 trial of patients with early-stage ALS. This development follows two late-stage trial failures reported by Novartis last week, plus the news that the pharma has paused multiple CAR T cell trials following patient deaths.

Original title: “Novartis halts TREM2 targeting ALS program in 4th trial stumble in a month”

atherosclerotic cardiovascular diseases increase als risk by 82 while optimal cardiovascular health halves it a 0 5 million prospective cohort study

Original title: “Atherosclerotic Cardiovascular Diseases Increase ALS Risk by 82% While Optimal Cardiovascular Health Halves It: A 0.5-Million Prospective Cohort Study.”

living with amyotrophic lateral sclerosis exploring what matters to patients and caregivers through their experiences

Original title: “Living with Amyotrophic Lateral Sclerosis: Exploring what matters to patients and caregivers through their experiences.”

emergencies in amyotrophic lateral sclerosis

Original title: “Emergencies in Amyotrophic Lateral Sclerosis.”

familial neuropathological and cellular analysis identify arpp21 as a major amyotrophic lateral sclerosis associated gene in french cohorts

Original title: “Familial, neuropathological and cellular analysis identify ARPP21 as a major amyotrophic lateral sclerosis associated gene in French cohorts.”

jp1 peptide modulates oxidative stress and autophagy via keap1 nrf2 are in als model mice

Original title: “JP1 peptide modulates oxidative stress and autophagy via Keap1-Nrf2-ARE in ALS model mice.”

bridging the gap neuroinflammation and the dawn of precision medicine in amyotrophic lateral sclerosis

Original title: “Bridging the gap: neuroinflammation and the dawn of precision medicine in amyotrophic lateral sclerosis.”

incidence and clinical characteristics of amyotrophic lateral sclerosis during the post covid 19 period a population based study from isfahan iran

Original title: “Incidence and clinical characteristics of amyotrophic lateral sclerosis during the post-COVID-19 Period: a population-based study from Isfahan, Iran.”

The Biotech Consortium to Accelerate ... therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI's mission is to keep American clinical trials in the U.S., ... The Biotech Consortium to Accelerate Innovation (BCAI) unites more than a dozen innovative biotechnology companies developing transformative therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI's mission is to keep American clinical trials in the U.S., so American patients have the earliest possible access to U.S.-developed medical innovation. CAMBRIDGE, Mass.--(BUSINESS WIRE)--A new survey reveals that U.S. biotechnology companies focused on developing treatments for rare and serious diseases overwhelmingly prefer to conduct first-in-human (FIH) clinical trials in the United States. However, historical obstacles posed by the U.S. Through strategic cost-saving initiatives, robust business partnerships, educational and networking opportunities, and proactive advocacy, MassBio empowers its members to launch the next generation of medical advancements to deliver the cures and therapies that enhance patient lives. ... 1U.S. Food and Drug Administration. Estimating the Maximum Safe Starting Dose in Initial Clinical Trials for Therapeutics in Adult Healthy Volunteers: Guidance for Industry. Instead, they are asking the FDA to implement the 10x safety margin as outlined in the Agency’s own 2005 guidance, Estimating the Maximum Safe Starting Dose in Initial Clinical Trials1. Restore predictable review timelines that reduce costly holds and rework. Strengthen sponsor-reviewer communication so trial design is driven by science, not procedural ambiguity. Ensure better alignment of review criteria with disease severity and life expectancy.

Original title: “New Survey Finds U.S. Biotechs Advancing Treatments for Rare and Serious Diseases Want to Run Clinical Trials in U.S. but Regulatory Hurdles are Sending Them Abroad”

lxr pathway activation by t0901317 a novel potential experimental strategy for als related cognitive and motor impairments via suppression of necroptosis associated ripk1 ripk3 mlkl markers

Original title: “LXR Pathway Activation by T0901317: A Novel Potential Experimental Strategy for ALS-Related Cognitive and Motor Impairments via Suppression of Necroptosis-associated RIPK1/RIPK3/MLKL Markers.”

clinical and genetic characteristics of 536 young onset amyotrophic lateral sclerosis in china a single center retrospective study

Original title: “Clinical and genetic characteristics of 536 young-onset amyotrophic lateral sclerosis in China: a single-center retrospective study.”

Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.

Original title: “Biogen’s targeted ALS treatment is reversing decline in some patients. Can more be helped?”

cocorv nf a powerful and cost effective tool for rare variant analysis leveraging external biobank sequence data identified new candidate predisposition genes in amyotrophic lateral sclerosis and neuroblastoma

Original title: “CoCoRV-nf: a powerful and cost-effective tool for rare variant analysis leveraging external biobank sequence data identified new candidate predisposition genes in amyotrophic lateral sclerosis and neuroblastoma.”

Globally, the annual incidence ... it a rare disease for which no fundamental cure currently exists.5 Multiple factors, including oxidative stress and glutamate-induced excitotoxicity, are involved in its pathophysiology, and treatment options to slow disease progression remain extremely limited. For this reason, ALS is recognized as a disease with a high level of unmet medical need, and the development and delivery of innovative therapies are strongly ... Globally, the annual incidence is estimated at approximately 1–2 cases per 100,000 population, making it a rare disease for which no fundamental cure currently exists.5 Multiple factors, including oxidative stress and glutamate-induced excitotoxicity, are involved in its pathophysiology, and treatment options to slow disease progression remain extremely limited. For this reason, ALS is recognized as a disease with a high level of unmet medical need, and the development and delivery of innovative therapies are strongly required. Shionogi & Co., Ltd. is a 148-year-old global, research-driven pharmaceutical company headquartered in Osaka, Japan, that is dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.” The company currently markets products in several therapeutic areas including anti-infectives, pain, and CNS disorders. and cardiovascular diseases. Shionogi’s research and development currently targets two therapeutic areas: infectious diseases and diseases with unmet medical needs in pain/CNS, including Alzheimer’s disease, oncology, rare diseases, and sleep apnea. Approved by the FDA in May 2022, RADICAVA ORS is an oral formulation that offers the same efficacy as the previously available intravenous (IV) RADICAVA® (edaravone).2,3 In a pivotal Phase 3 clinical trial of RADICAVA (n=68), treatment with edaravone slowed the loss of physical function by 33% compared with placebo (n=66) at 24 weeks, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).3,4 Common side effects reported include bruising (contusion), problems walking (gait disturbance), headache and fatigue. “Having an oral option has been helpful in shaping treatment plans and enabling more personalized, patient-centered decision-making.” · Shionogi remains committed to advancing the ALS community’s understanding of edaravone through continued education and the generation of critical data, including real-world evidence, biomarker research, and additional analyses from prior clinical trials.

Original title: “Shionogi Recognizes the Four-Year Anniversary of RADICAVA ORS® (edaravone) for Treatment of ALS - BioSpace”

tofersen treatment in sod1 als real world evidence from a retrospective multicenter study in france forsla study

Original title: “Tofersen Treatment in SOD1-ALS: Real-World Evidence from a Retrospective Multicenter Study in France (FORSLA Study).”

focal epilepsy expands the phenotype of fus associated amyotrophic lateral sclerosis

Original title: “Focal epilepsy expands the phenotype of FUS-associated amyotrophic lateral sclerosis.”

correction a rare missense variant impacting nek1 kinase function is associated with als

Original title: “Correction: A rare missense variant impacting NEK1 kinase function is associated with ALS.”

unraveling the role of lipid metabolism in als risk a mendelian randomization analysis using gwas data

Original title: “Unraveling the role of lipid metabolism in ALS risk: a Mendelian randomization analysis using GWAS data.”

effects of respiratory muscle training on respiratory function in patients with amyotrophic lateral sclerosis a systematic review and meta analysis

Original title: “Effects of respiratory muscle training on respiratory function in patients with amyotrophic lateral sclerosis: a systematic review and meta-analysis.”