riluzole / Generic mfg. - LARVOL DELTA

Riluzole and Sorafenib Tosylate in Treating Patients With Advanced Solid Tumors or Melanoma (clinicaltrials.gov) - P1 | N=35 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Sep 2026 ➔ Sep 2027

IO biomarker • Trial completion date • Cutaneous Melanoma • Melanoma • Oncology • Solid Tumor • BCL2 • BCL2L11

CORTEX-ALS: Efficacy and Safety of Cyclophosphamide in Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P2 | N=60 | Not yet recruiting | Sponsor: Huashan Hospital

New P2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Using machine learning to identify patient-ventilator asynchrony during non-invasive ventilation in Amyotrophic Lateral Sclerosis/Motor Neuron Disease (ERS 2026) - "Introduction: Non-invasive ventilation (NIV) confers three to four times more survival time than Riluzole, the most common medication, in Amyotrophic Lateral Sclerosis/Motor Neuron Disease (ALS/MND), but substantial barriers exist to optimal NIV access, uptake, and adherence... Our preliminary development work delivers a translational step towards automated PVA identification and classification for people with ALS/MND. Reducing manual clinician analysis should facilitate timelier and more effective NIV optimization and improve adherence, quality of life, and survival. We gratefully acknowledge funding support from FightMND, MRFF, and AEA Ignite."

Clinical • Machine learning • Non-invasive • Amyotrophic Lateral Sclerosis • CNS Disorders

Beneficial Effects of Putative Hydrogen Sulfide (H2S) Donor POM16 in a Genetic Model of Amyotrophic Lateral Sclerosis, FUS [1-359]-Transgenic Mice. (PubMed, Molecules) - "FUS [1-359]-tg mice, which recapitulate ALS syndrome, and their wild-type (WT) littermates received POM16 (at a dose of 50/mg/kg) or standard ALS therapy riluzole (at a dose of 8 mg/kg/day) dissolved in drinking water, or vehicle, for six weeks starting at nine weeks of age...Thus, new compound POM16 has a therapeutic potential to counteract ALS pathology that is likely mediated via anti-oxidative stress mechanisms. Given that any effective treatment of this devastating disease is currently lacking, it is hoped that POM16 can be a promising therapy for ALS."

Journal • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Inflammation • Oncology • FUS • IL1B • TNFA

Optimization and characterization of lipid-based nanocarriers for intranasal delivery in amyotrophic lateral sclerosis. (PubMed, Naunyn Schmiedebergs Arch Pharmacol) - "The optimized formulation exhibited sustained drug release, with approximately 84% drug release over 24 h, and remained stable for 60 days at room temperature. These findings suggest that riluzole-loaded nanostructured lipid carriers are a promising lipid-based nanocarrier system for intranasal delivery and further biological evaluation for nose-to-brain drug delivery in amyotrophic lateral sclerosis."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders

Safety, tolerability, and efficacy of RIPK1 inhibitor, SAR443820, in amyotrophic lateral sclerosis (HIMALAYA): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. (PubMed, Lancet Neurol) - P2 | "SAR443820 did not show clinical benefit and was associated with higher hepatic enzyme increase, indicating that further clinical development of SAR443820 in ALS is not warranted."

Clinical • Journal • P2 data • Amyotrophic Lateral Sclerosis • CNS Disorders • RIPK1

UV-programmed gelatin methacryloyl hydrogel microenvironments reveal a bell-shaped vascularization window and enable compartmentalized neuromuscular disease modeling. (PubMed, Biomaterials) - "Pharmacological treatment with the FDA-approved drug Riluzole partially restores these disease-associated phenotypes. Together, these findings establish UV-programmed GelMA hydrogels as an adaptable architectural-mechanical platform for identifying vascularization-permissive microenvironments and supporting compartmentalized neuromuscular disease modeling."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders

Neuroprotection of the Compressed Spine: A Systematized Narrative Review of Riluzole for Humans with Spinal Cord Injury. (PubMed, Neurosurg Clin N Am) - "Although primary efficacy endpoints were not uniformly met, multiple studies reported improved neurologic, functional, and patient-reported outcomes. Other agents with potential use or future promise are also highlighted."

Journal • Review • CNS Disorders • Orthopedics

Riluzole inhibits ovarian cancer cell migration by modulating extracellular matrix pathways (ECP 2026) - "RIL does not affect cell viability but significantly inhibits migration capacity in both cell lines, suggesting its possible use as an antimetastatic drug. These effects may be regulated by ITGB2 and other extracellular matrix-related mechanisms. Further studies are needed to validate these findings across multiple HGSOC cell lines, including chemotherapy-resistant cellular models."

Amyotrophic Lateral Sclerosis • CNS Disorders • High Grade Serous Ovarian Cancer • Oncology • Ovarian Cancer • Solid Tumor • ITGB2

An Adaptive, Time-Informed, Phenotype-Guided Pharmacological Framework for Severe Traumatic Brain Injury : A Hypothesis-Driven Narrative Review. (PubMed, J Korean Neurosurg Soc) - "Glibenclamide/glyburide and hypertonic sodium lactate produced preliminary imaging or physiological signals without demonstrated functional benefit. Riluzole was neutral in an adult randomized trial, whereas a small study in chronic TBI found that minocycline reduced a neuroinflammatory imaging signal but increased plasma neurofilament light. Metformin and N-acetylcysteine plus probenecid provided exploratory pharmacokinetic, biomarker, or safety data. Amantadine provided phenotype-specific clinical proof of principle in traumatic disorders of consciousness. Cerebrolysin showed mixed human evidence, including a favorable day-90 multidimensional ensemble signal that requires independent replication. Deferoxamine and clemastine remain preclinical...Module activation should require optimized foundational care, a matching phenotype, measurable target activation or engagement, and acceptable systemic safety. This framework prioritizes phenotype-enriched, single-module..."

Journal • CNS Disorders • Vascular Neurology • Plasma NfL

Therapeutic Challenges and Future Breakthroughs in Amyotrophic Lateral Sclerosis: From Precision Medicine to Innovative Trial Design. (PubMed, Drug Des Devel Ther) - "These include the modest survival benefit of riluzole, the narrow eligible population for edaravone, and the complex trajectory of sodium phenylbutyrate-taurursodiol, which received accelerated approval but was subsequently voluntarily withdrawn after its confirmatory Phase III trial failed to meet its primary endpoints. Future success depends on establishing multimodal biomarker panels, implementing genetic testing-guided individualized therapy, developing combination regimens, and integrating patient-reported outcomes with palliative care. Although substantial challenges remain, the clinical success of Tofersen provides evidence that precision therapeutic strategies may gradually transform ALS management toward a more individualized and disease-modifying approach."

Journal • Review • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • Inflammation • Palliative care

Modulating glutamate neurotransmission in in vivo models of Multiple Sclerosis: a narrative review. (PubMed, Front Immunol) - "Collectively, these data indicate that pharmacological modulation of glutamatergic signaling can engage multiple facets of EAE pathophysiology. However, completed clinical trials of repurposed glutamatergic drugs (riluzole, amantadine, memantine, lamotrigine) have so far failed to demonstrate disease-modifying efficacy in MS, underscoring a marked translational gap."

Journal • Preclinical • Review • CNS Disorders • Immune Modulation • Immunology • Inflammation • Multiple Sclerosis • TNFA

Trifunctional boron-based pyrazole increases survival, delays disease onset, and prevents ALS-induced cachexia in a humanized mouse model of Amyotrophic Lateral Sclerosis (ALS): Introducing BrosantrazoleTM (ACS-Fall 2026) - "Since the discovery of ALS in 1869, only two fully FDA-approved drugs are available: Edaravone (EDR), an antioxidant, and Riluzole (RZ), an anti-glutamatergic agent, with modest clinical benefits... A longitudinal preclinical study with a novel drug candidate, BSZ, demonstrated a satisfactory safety profile and significant preclinical, proof-of-concept efficacy in the SOD1 mouse model of ALS. Overall, these results validate the therapeutic potential of BSZ to increase survival, slow the progression of incurable ALS, and prevent ALS-induced weight loss. In addition, the current lead molecule, BSZ, is under patent, with a strong international search report from WIPO (PCT/CA2023/051352; WO/2024/103151), confirming its novelty, and industrial utility."

Preclinical • Amyotrophic Lateral Sclerosis • Cachexia • CNS Disorders • SOD1

Sex- and APOE-specific transcriptomic drug repurposing identifies four candidate therapeutics for Alzheimer's disease. (PubMed, Alzheimers Dement (N Y)) - "Drug reversal analysis identified four candidates: riluzole, chloroquine, acetazolamide, and anagrelide. Sex and APOE genotype define biologically distinct AD transcriptomic states, with subgroup-specific and shared pharmacologic reversibility. Integrating stratified transcriptomics with population-scale validation identified clinically actionable candidates supporting an AD precision drug repurposing framework."

Journal • Alzheimer's Disease • CNS Disorders • APOE

Identification of key genes related to arginine metabolism in immunoglobulin A nephropathy through the combination of scRNA-seq and bulk RNA-seq data. (PubMed, Front Immunol) - "Riluzole exhibited strong binding affinity for key genes and formed stable complexes...This study suggests that ARG1 and ARG2 may be key genes and potential mechanistic indicators of IgAN associated with AM, but it needs to be further verified in non-invasive samples and larger independent cohorts. Additionally, monocytes were recognized as key cells in the progression of IgAN, providing valuable insights to support the development of targeted therapies."

Journal • Chronic Kidney Disease • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease • ARG1 • ARG2 • CD8

Multidisciplinary clinic attendance and patterns of care in the U.S. National ALS Registry, 2013-2023. (PubMed, Amyotroph Lateral Scler Frontotemporal Degener) - "Attendees were more likely to use evidence-based interventions, including wheelchairs/scooters, noninvasive ventilation, percutaneous endoscopic gastrostomy (PEG), communication devices, and ALS disease-modifying therapies, specifically riluzole (72.7% vs. 48.1%) and edaravone (10.5% vs. 2.6%) (all p < 0.001)...For healthcare utilization, MDC attendance was associated with fewer emergency department visits among those with any use (RR 0.90, 95% confidence interval (CI) 0.82-0.99) and shorter hospital stays (β = -2.67 days, 95% CI -4.56 to -0.78). MDC attendance was associated with greater uptake of evidence-based interventions, higher rates of specialized care processes, and reduced healthcare utilization intensity, consistent with previously reported multidisciplinary ALS care outcomes."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Gastrointestinal Disorder

Lost in translation: why pharmacological trials keep failing in acute spinal cord injury. (PubMed, Arq Neuropsiquiatr) - "The current narrative review critically examines the seven agents evaluated in phase-II to -III clinical trials-methylprednisolone (MPSS), GM-1 monosialotetrahexosylganglioside, minocycline, riluzole, anti-Nogo-A antibody NG101, VX-210, and granulocyte colony-stimulating factor (G-CSF)-with emphasis on quantitative effect estimates, confidence intervals, and mechanisms of translational failure. The Nogo Inhibition in Spinal Cord Injury (NISCI) trial (2025), the most methodologically rigorous SCI trial to date, demonstrated no primary endpoint benefit but identified functional signals in motor-incomplete injuries. In total, six structural domains of translational failure are identified and methodological solutions proposed."

Journal • Review • CNS Disorders • Hematological Disorders • Infectious Disease • Novel Coronavirus Disease • Orthopedics • Respiratory Diseases

Prescribing and monitoring of riluzole in amyotrophic lateral sclerosis: a retrospective cohort study from Health New Zealand - Te Whatu Ora Waitaha Canterbury. (PubMed, N Z Med J) - "Adherence to recommended monitoring and Special Authority criteria for riluzole was inconsistent in this cohort. In the context of emerging evidence supporting broader survival benefit and reduced drug cost, further evaluation of current Special Authority prescribing criteria may be warranted."

Journal • Retrospective data • Amyotrophic Lateral Sclerosis • CNS Disorders

Huoling Shengji granule in amyotrophic lateral sclerosis: A multicenter, randomized, double-blind, riluzole-controlled trial. (PubMed, Neurotherapeutics) - "While the LOCF model showed no statistical difference, the MMRM analysis confirmed that HLSJ demonstrated significant efficacy superior to riluzole with a favorable safety profile. These findings provide a critical basis for conducting pivotal Phase III confirmatory trials of HLSJ for ALS treatment."

Clinical • Journal • Amyotrophic Lateral Sclerosis • CNS Disorders

Introducing Borsantrazole: A Trifunctional Boron-Based Pyrazole That Extends the Lifespan of Amyotrophic Lateral Sclerosis Mice. (PubMed, Adv Sci (Weinh)) - "Herein, we report that BSZ has demonstrated a favorable safety profile and compelling proof-of-concept efficacy in a ALS mouse model and has the potential to become a disease-modifying ALS therapeutic following further clinical development. Within a broader perspective of treatments for neurodegenerative diseases, BSZ offers a new paradigm for trifunctional small molecule targeting of oxidative stress that can mitigate neuronal deterioration and serve as a potential treatment."

Journal • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders • CA3 • CPLX2 • LRP4 • SQSTM1

Rapid Eye Movement Sleep Suppressing Antidepressant Prescription is Associated with Improved Survival in Amyotrophic Lateral Sclerosis. (PubMed, Neurol Sci) - "RSA prescription was associated with modestly better survival, but this association weakened markedly after matching and cannot establish causation; residual confounding, particularly by indication, cannot be excluded. These findings are hypothesis-generating, and prospective studies incorporating polysomnography and ALS-specific prognostic factors are needed."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Respiratory Diseases • Sleep Disorder

Comparative pH-Responsive Loading and Release of Structurally Distinct Central Nervous System Drugs from Graphene Oxide. (PubMed, ACS Appl Bio Mater) - "Herein, we present a comparative investigation of pH-responsive loading and release of two structurally distinct CNS drugs, d-cycloserine and riluzole, using graphene oxide as a common nanocarrier under identical experimental conditions...In vitro cytocompatibility studies using SH-SY5Y neuroblastoma cells demonstrated excellent biocompatibility for all formulations. This comparative study elucidates the critical role of drug chemistry in governing GO-drug interactions and provides design insights for graphene-based nanocarriers tailored for CNS drug delivery."

Journal • Neuroblastoma • Oncology • Solid Tumor

A Case of Bulbar-Onset Motor Neuron Disease Emerging in AChR-Positive Myasthenia Gravis (ICNMD 2026) - "Particular attention was paid to temporal evolution of phenotype, treatment responses, electrophysiological patterns and key features differentiating MG relapse from emerging ALS/MND, supported by a comparative table of bulbar-onset ALS versus bulbar MG. The patient achieved near-complete MG remission (MG-ADL 1) on prednisolone and rituximab but developed steroid toxicity and a non-tuberculous mycobacterial skin infection that constrained further immunosuppression...Repeat neurophysiology demonstrated preserved sensory responses, low-amplitude motor potentials without demyelination, normal repetitive nerve stimulation of limb and trapezius muscles, and widespread active and chronic denervation including tongue and paraspinals, findings incompatible with active MG and diagnostic of bulbar-onset ALS/MND. In MG patients, evolution to a non-fluctuating bulbar syndrome with cramps, fasciculations, upper motor neuron signs, limb wasting, respiratory decline and poor response..."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Gastrointestinal Disorder • Infectious Disease • Myasthenia Gravis • Nontuberculous Mycobacterial Disease • Ophthalmology • Pulmonary Disease • Respiratory Diseases

Repurposing Riluzole for Cancer-Related Cognitive Impairment: A Pilot Trial (clinicaltrials.gov) - P2 | N=75 | Recruiting | Sponsor: University of California, Irvine | N=51 ➔ 75 | Trial primary completion date: Dec 2026 ➔ Jul 2027

Enrollment change • Trial primary completion date • Bladder Cancer • Brain Cancer • Breast Cancer • Cognitive Disorders • Colorectal Cancer • Cutaneous T-cell Lymphoma • Dermatology • Gastric Cancer • Genito-urinary Cancer • Gynecologic Cancers • Head and Neck Cancer • Hematological Malignancies • Hodgkin Lymphoma • Kidney Cancer • Leukemia • Liver Cancer • Lung Cancer • Lymphoma • Melanoma • Multiple Myeloma • Mycosis Fungoides • Non-Hodgkin’s Lymphoma • Oncology • Ovarian Cancer • Pancreatic Cancer • Prostate Cancer • Renal Cell Carcinoma • Sarcoma • Solid Tumor

Increasing Prevalence of Amyotrophic Lateral Sclerosis in Austria Correlates With Reduced Mortality (ICNMD 2026) - "Although recent studies suggest that the global burden of the disease has increased over past decades, the trajectory of epidemiological metrics in Austria after 2011 remain unknown. Hospital discharge records and riluzole prescription databases were used to identify ALS cases between 2016 and 2023... In this study, we report on the epidemiology of ALS in Austria between 2016 and 2023. Notably, while the annual incidence remained stable over the study period, our findings indicate an increase of prevalence, most likely due to reduced mortality rates. Our results underscore the need for national healthcare policies to address the challenges posed by the growing disease burden."

Amyotrophic Lateral Sclerosis • CNS Disorders • Respiratory Diseases