PubMed Trending Research Digest — September 02, 2026

Automated digest · 100 articles · 15 research areas · September 02, 2026

Overview

This week’s digest is dominated by precision medicine themes: better patient stratification (e.g., asthma microbial signatures, eosinophilic IL-5 heterogeneity, IgA nephropathy pathology subtypes, and dynamic tumor immune microenvironment transitions), and more rational therapeutic design (target atlases for Ewing sarcoma, next-generation CAR-T “tonic signaling” tuning, and mechanistic mapping of resistance/evolution in cancers). Across multiple diseases, the emphasis is shifting from single biomarkers or static snapshots toward trajectories—how inflammation, immune states, or tumor biology change over time.

A second strong thread is mechanistic biology that connects cellular stress, metabolism, and immune function. Studies link ER stress to mitochondrial injury, RNA translation/epigenetic regulation to disease-relevant phenotypes, and mitochondrial metabolic fitness to NK-cell antitumor activity. In parallel, several cancer papers highlight how the tumor microenvironment and even intratumoral microbes can drive mutagenesis or immune suppression, suggesting new microbiome- or metabolism-targeted strategies.

Finally, the clinical translation angle shows up in consensus statements and real-world evidence: standardized procedural guidance (EUS gastroenterostomy), updated disease frameworks (heart failure definitions), and safety/monitoring efforts (pharmacovigilance for neuropathy/orthopedic signals; nephrotic syndrome case reports; complement-inhibitor era PNH algorithms). Together, these papers underscore a practical message: improving outcomes increasingly depends on combining mechanistic insight with standardized care pathways and robust safety surveillance.

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Asthma and Airway Microbiome

Whole-exome sequencing in individuals with obsessive-compulsive disorder and chronic tic disorders identifies 36 large-effect risk genes.

Whole-exome sequencing was performed in 3,964 individuals with obsessive-compulsive disorder (OCD), chronic tic disorders (CTDs), or both (including 2,418 trios) to identify rare, large-effect genetic risk variants. The study found an excess of de novo and rare protein-damaging mutations and identified 36 high-confidence risk genes (FDR < 0.1), including previously reported genes (CELSR3, CHD8, SCUBE1, WWC1) and additional OCD-overlapping loci (BRWD1, CELSR3, QRICH1, SYNE1). These results substantially expands the catalog of monogenic/large-effect contributors to OCD and CTDs and supports shared neurodevelopmental genetic architecture across these disorders.

Wang B, Tran MN, Wang S et al. · Nature neuroscience · (2026) · View on PubMed ↗

Development and external validation of a contrastive learning foundation model for ECG-based prediction of cardiovascular diseases and outcomes.

A contrastive-learning ECG foundation model (ECG-CLIP) was developed and externally validated using more than 1.7 million ECGs with clinician-overread reports from 542,288 patients in the Scripps Health GE MUSE system. The key finding was that ECG-CLIP improves generalisability and clinical applicability for ECG-based cardiovascular disease prediction compared with traditional task-specific machine-learning approaches that rely heavily on labeled outcomes. This provides a scalable, label-efficient framework for more robust ECG interpretation that could improve downstream diagnostic and prognostic performance across diverse clinical settings.

Ko M, Gadaleta M, Topol EJ et al. · The Lancet. Digital health · (2026) · View on PubMed ↗

RANDOMIZED CONTROLLED STUDY OF NEMOLIZUMAB IN PATIENTS WITH CHRONIC KIDNEY DISEASE AND ASSOCIATED PRURITUS: NIKAIA-1.

In a 12-week randomized, placebo-controlled trial, nemolizumab (IL-31 monoclonal antibody; 30 mg or 60 mg every 4 weeks) was tested in 258 hemodialysis patients with chronic kidney disease-associated pruritus (CKD-aP). The study assessed safety and efficacy on pruritus (including Worst Itch Numerical Rating), sleep disturbance, and quality of life, with the primary endpoint defined as the proportion achieving at least a 4-point improvement in Worst Itch-Numerical Rating. If the reported results confirm meaningful itch reduction with acceptable safety, nemolizumab would represent a targeted systemic option for refractory CKD-aP in hemodialysis patients.

Mathur VS, Fishbane S, Ständer S et al. · Journal of the American Academy of Dermatology · (2026) · View on PubMed ↗

Adolescent health across Asia Pacific, 2000-23: a systematic analysis for the Global Burden of Disease Study 2023.

Using Global Burden of Disease Study 2023 data, this systematic analysis quantified adolescent health burden, mortality, and prevalence of adolescent risk factors across the Asia Pacific region from 2000–2023. The key finding is a comprehensive, subregional and national mapping of leading contributors to disease and risk among adolescents aged 10–24 years. This evidence base is intended to guide more targeted public-health interventions and policy planning for adolescent health in Asia Pacific countries.

The Lancet. Child & adolescent health · (2026) · View on PubMed ↗

Live-cell transcriptomics with engineered virus-like particles.

Engineered virus-like particles (VLPs) were used to enable live-cell transcriptomics by having mammalian cells export mRNA in VLPs, allowing repeated sampling of culture media without lysing cells. The key finding was that VLP-based sampling faithfully captured evolving transcriptional states during acute inflammatory stimulation of primary cell spheroids and across multi-day differentiation of pluripotent stem cells. This technique enables longitudinal measurement of transcriptional dynamics in the same biological system, expanding experimental options for studying time-dependent gene regulation.

Najia MA, Le A, Borrajo J et al. · Cell · (2026) · View on PubMed ↗

Cross-species single-cell atlas of the striatum defines cell-type and subregion disease vulnerabilities.

Single-nucleus RNA sequencing was used to build a cross-species striatum atlas from 109 human and 22 mouse samples spanning dorsal and ventral striatum to define cell-type and subregion disease vulnerabilities. The study identified rare neuronal subpopulations and transcriptional gradients along the dorsolateral-to-ventromedial axis, with notable species differences in these patterns. These findings help pinpoint which striatal cell states and subregions may be preferentially vulnerable in neurodegenerative and neuropsychiatric disorders and improve translational targeting across models.

Linville RM, James BT, Galani K et al. · Cell · (2026) · View on PubMed ↗

Tumor immune microenvironment remodeling predicts response to checkpoint inhibitor therapy.

A longitudinal single-cell RNA sequencing atlas comprising 441 samples from 241 patients across ten cancer entities was analyzed to determine how tumor immune microenvironment (TIME) remodeling relates to response to immune checkpoint inhibitor (ICI) therapy. The key finding was that ~40% of tumors shifted between conserved TIME states during treatment and that the transition (rather than baseline state) better predicted outcomes, with favorable transitions toward inflamed or B cell-enriched subtypes associated with improved response. This supports dynamic TIME state changes as a clinically actionable biomarker for ICI responsiveness.

Lin Z, Chandra M, Srinivas N et al. · Cancer cell · (2026) · View on PubMed ↗

New Evidence in Heart Failure: 2026 Update.

This 2026 update synthesized current evidence on heart failure (HF) epidemiology, prognosis, and disease trajectories, including concepts such as improvement, remission, and recovery, alongside updates to HF classification. The key finding is that the Second Universal Definition of HF updates the framework beyond traditional ejection-fraction-based categorization. Clinically, this helps standardize diagnosis and classification and informs how emerging trajectory-based concepts may be incorporated into HF management and research.

Liguori V, Rizzello A, Adamo M et al. · ESC heart failure · (2026) · View on PubMed ↗

Prevalence of Slowly Expanding Lesions in Patients With Multiple Sclerosis: A Systematic Review and Meta-Analysis.

A systematic review and meta-analysis estimated the prevalence of slowly expanding lesions (SELs) among T2 lesions and among patients with at least one SEL in multiple sclerosis (MS), and quantified overlap with paramagnetic rim lesions (PRLs). The key finding was the pooled proportion of SELs and the degree to which SELs coincide with PRLs on susceptibility-sensitive MRI, clarifying how these chronic active lesion phenotypes relate. This informs MRI-based risk stratification and may refine understanding of chronic inflammation mechanisms in MS.

Liampas A, Artemiadis A, Tseriotis VS et al. · Neurology · (2026) · View on PubMed ↗

AML1-ETO hijacks a distal enhancer of NAT10 to reprogram glutathione metabolism and sustain leukemia stem cell stemness.

In t(8;21) acute myeloid leukemia (AML) models, the study mapped AML1-ETO and H3K27ac CUT&Tag landscapes in primary CD34+ cells and AML cell lines to identify regulatory DNA elements involved in oncogenic reprogramming. The key finding was that AML1-ETO binds a distal enhancer of the RNA ac4C writer NAT10, driving NAT10 transcription and reprogramming glutathione metabolism to sustain leukemia stem cell (LSC) stemness. This mechanistic link between AML1-ETO enhancer hijacking and redox/metabolic control highlights NAT10 as a potential therapeutic vulnerability in AML1-ETO–driven leukemia.

Li S, He S, Su Y et al. · Proceedings of the National Academy of Sciences of the United States of America · (2026) · View on PubMed ↗

Mycobacterial vaccination of fish and human tuberculin skin test reactivity: a testable One Health interface.

This article reviewed and proposed a testable One Health framework linking mycobacterial vaccination strategies in fish with interpretation of tuberculin skin test (TST) reactivity in humans and livestock. It highlights that TST readouts using purified protein derivative (PPD) are confounded by BCG vaccination, exposure to environmental non-tuberculous mycobacteria (NTM), chosen cutoff thresholds, and local epidemiology, and it outlines how fish vaccination could be incorporated into cross-species surveillance. The significance is improved, more interpretable TST-based monitoring of mycobacterial immune sensitization across human–animal–environment interfaces.

Mataragka A · Future microbiology · (2026) · View on PubMed ↗

Guidelines for diagnostic testing in adults with presumed atopic dermatitis refractory to treatment.

This work developed evidence-informed clinical guidelines for diagnostic testing in adults with presumed atopic dermatitis (AD) who are refractory to optimized treatment. It addresses the need to reassess diagnosis and evaluate alternative or concomitant conditions using a structured, multidisciplinary approach grounded in GRADE methodology. The clinical significance is standardized diagnostic workup to reduce misdiagnosis and improve management of treatment-refractory adult AD.

Sidbury R, Wu PA, Alikhan A et al. · Journal of the American Academy of Dermatology · (2026) · View on PubMed ↗

Case Report: Effective IL-4/IL-13 axis suppression and resolution of severe atopic dermatitis by dupilumab monotherapy in a patient with IPEX syndrome.

This case report studied dupilumab monotherapy in a 4-year-old boy with IPEX syndrome caused by a hemizygous FOXP3 missense variant (c.1150G>A; p.Ala384Thr). Dupilumab targeting the IL-4/IL-13 axis led to resolution of severe atopic dermatitis with corresponding clinical and immunologic improvement. The finding is significant because it suggests a potential non-immunosuppressive, targeted option for type 2 inflammation in FOXP3-related IPEX, where prior reports used dupilumab only as combination therapy.

Alshanti M, MacDougall MS, Gokbak MN et al. · Frontiers in immunology · (2026) · View on PubMed ↗

Long-term adverse effects following COVID-19 vaccination: A comparative analysis of Oxford AstraZeneca, Pfizer-BioNTech, and Moderna vaccines.

This cross-sectional study assessed long-term adverse clinical symptoms after COVID-19 vaccination with Oxford AstraZeneca (ChAdOx1 nCoV-19), Pfizer-BioNTech (BNT162b2), or Moderna (mRNA-1273) in 429 participants recruited via an online Google Form. Participants reported the most common long-term adverse effects across vaccine groups, enabling comparative characterization of persistent symptom patterns by vaccine type. The results are relevant for post-authorization pharmacovigilance and for counseling patients about longer-term tolerability after different vaccine platforms.

Aldali JA, Meo SA · Medicine · (2026) · View on PubMed ↗

From Pustules to Pruritus: A Novel IL36RN Frameshift Variant Reveals a Triple Biologic Paradox and a Th17-To-Th2 Phenotypic Transition.

This case report studied a 64-year-old woman with longstanding psoriasis vulgaris and atopic comorbidities who developed generalized pustular psoriasis after biologic treatments, followed by transition to chronic eczematous dermatitis. Genetic testing identified a novel heterozygous IL36RN frameshift variant (c.319del; p.Leu107Serfs65), and the patient’s phenotype shifted from a Th17-associated pustular pattern to a Th2-like eczematous pattern over subsequent months. The “triple biologic paradox” highlights how IL36RN loss-of-function variants can drive sequential inflammatory phenotypes, informing genetic evaluation and biologic risk considerations in complex psoriasis–atopy overlap.

Çalıcıoğlu F, Çalıcıoğlu N, Doğan ME et al. · The Journal of dermatology · (2026) · View on PubMed ↗

Phase separation in neurodegenerative disorders: a metabolic perspective on protein aggregation and therapeutic targeting.

This review studied how liquid–liquid phase separation (LLPS) and metabolic context influence protein aggregation in neurodegenerative disorders involving TDP-43, FUS, tau, and α-synuclein. It highlights that metabolic state—such as ATP availability and redox/NAD+/NADH balance—can regulate condensate assembly, material properties, and liquid-to-solid maturation, thereby shaping aggregation and pathology. The metabolic perspective is significant because it suggests new therapeutic targeting strategies that modulate cellular energetics and condensate dynamics rather than only protein-intrinsic features.

Peng S, Chen H, Yin G et al. · Translational neurodegeneration · (2026) · View on PubMed ↗

Short-term effects of combinations of heart failure therapies on blood pressure, kidney function and serum potassium.

This pooled individual participant-level analysis studied short-term (2–12 week) effects of combination heart failure therapies on systolic/diastolic blood pressure, kidney function (eGFR), and serum potassium in 38,753 participants across HFrEF and HFmrEF/HFpEF. The authors developed a prediction model estimating these short-term treatment effects to address concerns about hypotension, renal dysfunction, and hyperkalemia. This is clinically significant because it supports safer initiation and titration of comprehensive guideline-directed medical therapy by forecasting early physiologic responses.

Wang N, Claggett BL, Pfeffer MA et al. · Nature medicine · (2026) · View on PubMed ↗

Neutrophil serine proteases inhibit thermogenic capacity of visceral white adipose tissue.

This study examined how neutrophil infiltration and neutrophil serine proteases (NSPs)—neutrophil elastase (NE) and proteinase 3 (PR3)—affect visceral white adipose tissue browning and thermogenesis in male mice. Genetic deletion or local pharmacologic inhibition of NE with sivelestat rescued visceral fat browning and enhanced thermogenesis by suppressing beige adipogenesis via CDK4/cyclin D1 downregulation and cell-cycle arrest. The results suggest NE/PR3 as actionable drivers of impaired energy metabolism in obesity and as potential targets to restore thermogenic capacity.

Yuan L, Wu X, Zong J et al. · Nature metabolism · (2026) · View on PubMed ↗

Choice of hypomethylating agent for newly diagnosed TP53-mutant acute myeloid Leukemia: a COMMAND registry study.

This multicenter retrospective COMMAND registry study compared induction choices—decitabine (DEC) versus azacitidine (AZA), including venetoclax (VEN) combinations—in newly diagnosed TP53-mutant acute myeloid leukemia (TP53-MT AML). Baseline clinical/genomic features were similar between groups, and the study evaluated whether DEC achieved superior outcomes such as deeper TP53 mutation clearance and response rates compared with AZA in this specific TP53-MT population. Clinically, the work aims to guide selection of hypomethylating agents for TP53-MT AML induction strategies, potentially optimizing response depth and durability.

Badar T, Foran J, Jamy O et al. · Leukemia · (2026) · View on PubMed ↗

Incidence, prevalence, and survival outcomes of patients with myeloproliferative neoplasms in the United States: a Surveillance, Epidemiology, and End Results (SEER) database analysis, years 2000-2021.

This SEER-17 database analysis studied incidence, prevalence, and survival outcomes of adult myeloproliferative neoplasms (MPNs)—polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis (PMF), and chronic myeloid leukemia (CML)—from 2000 to 2021. Using 63,242 patients (ET, PV, PMF, and CML cohorts), the authors quantified contemporary epidemiology and survival patterns across these MPN subtypes. These updated population-level estimates are significant for benchmarking disease burden and informing prognosis and healthcare planning as diagnostics and therapies evolve.

Singhal S, Mishra R, Arora S et al. · Leukemia · (2026) · View on PubMed ↗

Tisagenlecleucel for post-transplant relapse in young acute lymphoblastic leukemia patients: European real-world determinants of outcome.

This European real-world multicenter study evaluated determinants of outcome for tisagenlecleucel (tisa-cel) in children and young adults with B-cell acute lymphoblastic leukemia (B-ALL) who relapsed after allogeneic hematopoietic stem cell transplant (HSCT). In 220 patients, 2-year event-free survival was 43.6% and overall survival was 67.2%, with CAR-T failure occurring in 57.1% of cases; donor source influenced outcomes, with matched sibling donor (MSD) relapse showing lower 2-year OS and higher CAR-T failure incidence than alternative donors. The findings help refine risk stratification and expectations for CAR-T performance after post-transplant relapse based on transplant donor context.

Moser LM, Hutter M, Ahlmann M et al. · Leukemia · (2026) · View on PubMed ↗

Immune-mediated rheumatoid arthritis: from single-cell genomics to new therapies.

This Nature Immunology Perspective reviewed how single-cell genomics has advanced understanding of immune-mediated rheumatoid arthritis (RA) and how to translate descriptive datasets into mechanistic and therapeutic insights. It highlights emerging approaches—spatial transcriptomics, ex vivo mechanistic and organoid studies, and improved human-data-informed animal models—to move from correlation to causality in identifying pathogenic lymphocyte, myeloid, and fibroblast subsets. The perspective is significant for guiding next-generation RA research strategies that can yield actionable targets and therapies.

Ivashkiv LB · Nature immunology · (2026) · View on PubMed ↗

Monocyte-derived galectin-1hi cells provide innate immune help in the generation of functional memory CD8+ T cells.

This study investigated how monocyte-derived galectin-1hi cells contribute to the generation of functional memory CD8+ T cells during influenza infection. CCR2+ monocytes differentiated into memory-stage CCR2-tdTomato+ cells that persisted in the lung for over 4 months, and selective depletion of these memory-stage cells reduced lung CD8+ tissue-resident memory (TRM) formation and impaired secondary heterosubtypic protection. The work identifies a specific monocyte-derived population as an innate immune “helper” for durable TRM-mediated antiviral immunity.

Lim K, Dahal A, Lv X et al. · Nature immunology · (2026) · View on PubMed ↗

Large language models decode narrative pathology reports to define clinically relevant subtypes in IgA nephropathy.

This study assessed whether large language models (LLMs) can extract clinically relevant pathology information from routine narrative biopsy reports to stratify IgA nephropathy (IgAN) risk. In 3,078 adults with primary IgAN from two hospitals in Wenzhou, China, LLM-based analysis identified reproducible pathological subtypes with prognostic relevance beyond structured Oxford MEST-C scoring. The approach is significant because it leverages unstructured clinical text to improve biopsy-based risk stratification in a heterogeneous disease.

Zhang J, Lu J, Jiang L et al. · Nature communications · (2026) · View on PubMed ↗

Molecular mechanism of pore formation by Plasmodium Perforin-like Protein 2.

This study investigated the molecular mechanism of pore formation by the Plasmodium perforin-like protein 2 (PLP2), focusing on Plasmodium vivax PLP2-mediated membrane attack. Using cryo-electron microscopy and tomography, the authors showed that PvPLP2 assembles on lipid bilayers into heterogeneous arc- and ring-shaped pores, including a resolved 17-subunit pore complex in which MACPF domains form the central β-barrel. These structural insights are significant for understanding how PLP2 drives erythrocyte rupture and for informing potential antimalarial strategies targeting pore formation.

Zhang Y, Zhong L, Song Y et al. · Nature communications · (2026) · View on PubMed ↗

Deep-learning-enabled multi-omics analyses for prediction of future metastasis in cancer.

This study developed and evaluated EmitGCL, a deep-learning framework for predicting future metastasis and associated biomarkers from multi-omics data across multiple cancer types. EmitGCL outperformed other computational tools across six cancer types from seven patient cohorts and could identify occult metastatic cells in a lymph node-negative breast cancer patient who appeared disease-free on conventional imaging. The method is clinically significant because it offers a more sensitive, biomarker-linked way to forecast metastasis risk before it becomes detectable by standard imaging.

Wang X, Duan M, Snyder AJ et al. · Nature communications · (2026) · View on PubMed ↗

The airway microbiome in asthma.

This review studied evidence linking the airway microbiome (including bacterial dysbiosis and emerging roles for the airway virome and mycobiome) to asthma development, severity, and inflammatory endotypes in human patients. It found that airway microbiome dysbiosis is associated with asthma and may relate to inflammatory endotypes, while the directionality (whether dysbiosis drives inflammation or results from it) remains unresolved. Understanding these microbial signatures could improve asthma phenotyping and guide future microbiome-targeted prevention or treatment strategies.

Richardson H, Pollock J, Chan R et al. · Chest · (2026) · View on PubMed ↗

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Eosinophilic Inflammation and IL-5 Pathways

IL-5 Biology and Eosinophil Regulation: Advances, Resistance Pathways and Future Directions.

This narrative review examined IL-5 biology and eosinophil regulation, focusing on why responses to IL-5/IL-5Rα–targeted biologics in severe eosinophilic asthma are heterogeneous. It found that persistent symptoms after anti–IL-5 or anti–IL-5Rα therapy may reflect ongoing eosinophilic inflammation, partial response, comorbid residual disease, or IL-5–independent inflammatory mechanisms. These insights are clinically important for refining patient selection, resistance-pathway understanding, and future therapeutic development in eosinophil-driven airway disease.

Patella V, Nicoletta C, Ferrara F et al. · Respiratory medicine · (2026) · View on PubMed ↗

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Lung Cancer Therapies and Pulmonary Toxicity

Pulmonary Toxicities of Antibody-Drug Conjugates in Small Cell and Non-Small Cell Lung Cancer: A Systematic Review and Meta-analysis.

This systematic review and meta-analysis studied pulmonary toxicities of antibody-drug conjugates (ADCs) in patients with small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC). It aimed to quantify the incidence and severity of pulmonary adverse events and compare toxicity differences by tumor type and ADC drug-design features. The findings are significant for improving safety monitoring and risk–benefit decisions as ADCs expand in lung cancer care.

Paredes de la Fuente R, Borea R, Enrico D et al. · Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer · (2026) · View on PubMed ↗

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Drug Safety Pharmacovigilance (Neuropathy/Orthopedic/Other)

Niche-Mediated Neural Priming Enables Robust and Scalable Generation of Human Choroid Plexus Organoids.

This study investigated how stem-cell niche “neural priming” improves generation of human choroid plexus (ChP) organoids from human embryonic stem cells (hESCs). Preconditioning hESCs by switching culture from E8 to mTeSR1 medium increased ChP organoid induction efficiency from <3% to ~68%, with mechanistic suppression of BMP and WNT signaling. The scalable niche-aware protocol provides a practical human ChP model for studying blood–CSF barrier biology and CSF-related development and disease.

Chen Z, Jiang L, Wang X et al. · Advanced science (Weinheim, Baden-Wurttemberg, Germany) · (2026) · View on PubMed ↗

Real-World Safety Signals of Fluoroquinolone-Associated Peripheral Neuropathy: Analysis of US and Canadian Databases Combined with Mechanistic Exploration.

This pharmacovigilance study analyzed peripheral neuropathy signals associated with fluoroquinolones—ciprofloxacin, levofloxacin, moxifloxacin, and ofloxacin—in adverse event reports from the FDA Adverse Event Reporting System (FAERS, 2004–2025) and the Canada Vigilance Adverse Reaction Database (CVARD). Using disproportionality signal detection algorithms (ROR, PRR, BCPNN, and empirica), it characterized PN safety signals and explored potential mechanistic explanations. The results are clinically important for antimicrobial prescribing and for identifying patients at risk of fluoroquinolone-associated neuropathy.

Guo B, Zou Z, Lu J et al. · International journal of antimicrobial agents · (2026) · View on PubMed ↗

Atypical radiographic changes seen in dogs treated with bedinvetmab (Librela).

This veterinary pharmacovigilance study investigated musculoskeletal adverse events and atypical radiographic changes in dogs treated with bedinvetmab (Librela) using the Zoetis Global Pharmacovigilance database. It found and categorized adverse event reports with radiographs/radiology reports, including cases consistent with osteoarthritis progression and other specific orthopedic findings. The results are significant for clinicians monitoring musculoskeletal safety signals in dogs receiving bedinvetmab.

Budsberg SC, Clements DN, Regan DP et al. · Journal of the American Veterinary Medical Association · (2026) · View on PubMed ↗

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Neurodegeneration and ALS (Glia, ER-mito Crosstalk, Protein Strains)

Seeds from ALS patients homozygous for the SOD1 D90A mutation transmit two types of SOD1 aggregation and motor neuron disease.

This study investigated prion-like propagation of human SOD1 (hSOD1) aggregation by using seed preparations derived from ALS patients homozygous for the SOD1 D90A mutation. It found that these patient-derived seeds transmit two structurally distinct SOD1 aggregation strains—strain A and strain B—consistent with strain-specific aggregate formation observed in hSOD1 transgenic mouse models, with D90A homozygotes producing a distinct strain B while also generating strain A. The significance is stronger evidence that human SOD1 D90A ALS involves strain-like, transmissible aggregate conformations that may underlie phenotype variability.

Henne C, Sigfridsson I, Brännström T et al. · Acta neuropathologica · (2026) · View on PubMed ↗

Selective targeting of the oligodendroglial GPR17 receptor improves myelin integrity and motor function in female SOD1G93A mice.

This preclinical study tested selective pharmacologic targeting of the oligodendroglial GPR17 receptor in female SOD1G93A mice, a model of amyotrophic lateral sclerosis (ALS). It found that pathological GPR17 upregulation marks an immature oligodendroglial state and that GPR17 targeting improved myelin integrity and motor function in vivo. These findings are significant because they validate GPR17 as a druggable target for potential disease-modifying strategies in ALS.

Raffaele S, Bonifacino T, Mannella FC et al. · Pharmacological research · (2026) · View on PubMed ↗

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Cell Stress and Organelle Crosstalk (UPR–Mitochondria, Condensates, RNA Aggregation)

Multi-organ single-cell transcriptomic atlas identifies QrIAA14 as a candidate negative regulator of adventitious root development in Quercus robur.

This study generated a multi-organ single-cell RNA-seq atlas of leaf, stem, and root tissues in the woody plant Quercus robur to define cellular programs controlling adventitious root development. The authors identified QrIAA14 as a candidate negative regulator of adventitious root development based on cell-type–resolved transcriptional trajectories and organ-specific gene expression patterns. Scientifically, it provides a cellular-resolution regulatory target (QrIAA14) for improving vegetative propagation efficiency in oak.

Hui W, Li J, Li H et al. · PLoS genetics · (2026) · View on PubMed ↗

Unbiased screen of human transcriptome reveals an unexpected role of 3’UTRs in translation initiation.

This study developed a circRNA-based, unbiased screen to identify cap-independent translation initiators (CiTIs) across the human transcriptome. It found that most CiTIs reside in 3’UTRs and promote translation initiation of mRNAs with highly structured 5’UTRs by recruiting eIF3 and DHX29 to unwind 5’UTR structures and enable ribosome scanning. These findings reveal an unexpected 3’UTR-driven mechanism for cap-independent translation that could inform therapeutic control of translation in diseases with altered 5’UTR structure.

Yang Y, Fan X, Ye Y et al. · Nature communications · (2026) · View on PubMed ↗

Crosstalk between UPR and mitochondria: The Triad of ER-Mitochondria Contacts, Ca²⁺, and ROS.

This mechanistic review studied how unfolded protein response (UPR) signaling communicates with mitochondria through ER–mitochondria contact sites (ERMCs), Ca2+ handling, and reactive oxygen species (ROS). It highlighted the CHOP–ERO1A–IP3R axis as a key pathway for recruiting mitochondria during adaptive UPR, while sustained activation can become maladaptive via outer mitochondrial membrane permeabilization and mitochondrial dysfunction. The work is significant for understanding how ER stress transitions from protective signaling to mitochondrial injury, informing therapeutic targeting of ER–mitochondria crosstalk.

Zito E, Hajnóczky G · Pharmacological research · (2026) · View on PubMed ↗

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Gastroenterology Procedures and GI Surgical/Endoscopic Standards

Standard reporting on duodenal stump leakage after gastric cancer resections: multicentre analysis from the GASTRODATA Study Group (the STARDUST study).

This multicentre retrospective cohort analysis (STARDUST/GASTRODATA) studied the occurrence rate and management strategies for duodenal stump leakage (DSL) after gastric cancer gastrectomy. Using data from 24 high-volume European centers covering surgeries from 2019 to 2022, it aimed to characterize DSL incidence and identify factors associated with treatment strategy decisions. The significance is improved international benchmarking and evidence to guide postoperative risk management for a major cause of mortality after gastrectomy.

Molteni M, Puccetti F, Baiocchi GL et al. · The British journal of surgery · (2026) · View on PubMed ↗

Endoscopic Ultrasound-guided Gastroenterostomy: An International Consensus on Current and Expanding Indications, Techniques, Periprocedural Care and Institutional Requirements.

This international consensus statement studied endoscopic ultrasound-guided gastroenterostomy (EUS-GE) and related EUS-guided anastomoses across heterogeneous indications, techniques, and peri-procedural care. Using a modified Delphi process with evidence synthesis and GRADE assessment, it produced standardized recommendations for current and expanding indications, procedural/technical standards, and institutional requirements. The consensus is clinically important for improving safety, quality, and consistency of EUS-GE adoption for gastric outlet obstruction and complex GI scenarios.

Vanella G, Neesse A, Binda C et al. · Endoscopy · (2026) · View on PubMed ↗

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COVID-19 Therapeutics and Post-Acute Outcomes

Proton pump inhibitors in invasively ventilated patients with SARS-CoV-2: a substudy of the re-evaluating the inhibition of stress erosions trial.

This pre-planned substudy of the randomized REVISE trial studied pantoprazole effects in invasively ventilated adults with SARS-CoV-2 by comparing outcomes with a propensity-matched non-infected cohort and testing whether treatment effects differed by infection status. It assessed clinical characteristics and outcomes in mechanically ventilated patients receiving pantoprazole, stratified by SARS-CoV-2 infection status. The results are clinically significant because they address whether acid suppression with a proton pump inhibitor worsens or modifies outcomes in severe COVID-19 critical illness.

Dennis B, Heels-Ansdell D, Ibrahim Q et al. · BMJ open · (2026) · View on PubMed ↗

Nirmatrelvir-ritonavir targeting viral persistence in post-COVID-19 condition (long COVID) in the USA (RECOVER-VITAL): a randomised, double-blind, placebo-controlled, phase 2 trial.

This randomized, double-blind, placebo-controlled phase 2 trial studied whether the SARS-CoV-2 antiviral combination nirmatrelvir–ritonavir reduces long COVID symptoms in adults with persistent symptoms (≥12 weeks) across cognitive, autonomic, or exercise phenotypes at 69 US sites. It tested the viral persistence hypothesis by comparing symptom outcomes between nirmatrelvir–ritonavir and placebo. The trial is clinically important because it evaluates a targeted antiviral strategy for a major unmet need in post-acute COVID-19 care.

Baden LR, Shah NS, Liu STH et al. · The Lancet. Infectious diseases · (2026) · View on PubMed ↗

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Cardiology: Heart Failure and Pacing/Resynchronization

Catheter-Directed Thrombolysis in Intermediate-High-Risk Pulmonary Embolism.

This multicenter, open-label randomized trial studied catheter-directed thrombolysis with alteplase plus anticoagulation versus anticoagulation alone in patients with intermediate-high-risk acute pulmonary embolism defined by simplified Pulmonary Embolism Severity Index (sPESI) ≥1 plus right ventricular dysfunction and elevated cardiac troponin or natriuretic peptide. Catheter-directed alteplase improved clinical outcomes compared with standard anticoagulation alone (trial results reported in the full text). If confirmed, this supports using catheter-directed alteplase in intermediate-high-risk PE to reduce deterioration and improve prognosis beyond anticoagulation alone.

Kroupa J, Radvan M, Mrozek J et al. · The New England journal of medicine · (2026) · View on PubMed ↗

Inhibition of Programmed Cell Death-1 in Cytotoxic CD8+ T Cells Exacerbates Pressure Overload-Induced Cardiac Injury.

This preclinical study tested whether blocking programmed cell death-1 (PD-1) in cytotoxic CD8+ T cells worsens pressure overload–induced cardiac injury using transverse aortic constriction (TAC) in mice. Anti–PD-1 antibody treatment exacerbated TAC-induced cardiac remodeling and heart failure, and the authors used T cell-, myeloid-, and CD8+ T cell-specific Pdcd1 knockouts plus Cxcr3 and granzyme B (Gzmb) knockout models with flow cytometry, Western blotting, immunofluorescence, and bulk RNA-seq to define mechanisms. The findings provide a mechanistic explanation for the increased HF risk seen with anti–PD-1 therapy in susceptible patients and suggest pathways (e.g., CXCR3/granzyme B) to mitigate harm.

Wu MM, Sun Y, Zhang Y et al. · Circulation · (2026) · View on PubMed ↗

POC1A promotes the proliferation, metastasis and stemness of bladder cancer by stabilizing BMI1 via USP7.

This study investigated the role of POC1A in advanced bladder cancer (BLCA) and its molecular interaction partners in promoting tumor progression. POC1A was overexpressed in BLCA and its knockdown inhibited BLCA cell proliferation, metastasis, and stemness by stabilizing BMI1 through the deubiquitinase USP7 (POC1A–USP7–BMI1 complex). These findings identify the POC1A/USP7/BMI1 axis as a mechanistic therapeutic target to limit aggressive BLCA phenotypes.

Wei H, Bai R, Liu J et al. · Oncogene · (2026) · View on PubMed ↗

Targeted left ventricular lead placement in biventricular pacing for heart failure: a national, multicentre, double-blind, randomised controlled trial in Denmark.

This national, multicentre, double-blind, randomized controlled trial studied targeted left ventricular lead placement in biventricular pacing for heart failure in Danish patients with wide QRS on guideline-directed medication. It tested whether placing the left ventricular lead at the site of latest electrical activation improves outcomes versus a control placement strategy. The results are significant for optimizing cardiac resynchronization therapy programming to improve clinical outcomes in eligible heart failure patients.

Nielsen JC, Svendsen JH, Johansen JB et al. · Lancet (London, England) · (2026) · View on PubMed ↗

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Ophthalmology: Cataract and Eye Care Standards

Performance of Photon-counting CT for Assessing Pretreatment Breast Cancer: Comparison with Mammography, MRI, and 18F-FDG PET/CT.

This prospective study evaluated photon-counting CT (PCCT) for pretreatment breast cancer assessment by comparing it with mammography, MRI, and 18F-FDG PET/CT. Female participants with breast lesions (BI-RADS 4C or higher) underwent multiphasic contrast-enhanced PCCT and MRI, with FDG PET/CT performed in a subset, to assess feasibility and diagnostic performance. Clinically, it supports the potential role of PCCT as an improved imaging modality for initial breast cancer characterization prior to therapy.

Fu Q, Zeng Y, Chang R et al. · Radiology · (2026) · View on PubMed ↗

ESCRS guideline for cataract surgery 2024: executive summary.

This ESCRS guideline executive summary studied evidence-based cataract surgery care pathways in patients and clinicians, addressing 32 key clinical questions using the GRADE framework. It recommends intracameral antibiotics to reduce endophthalmitis risk, topical anesthesia as the preferred technique, and toric intraocular lenses for corneal astigmatism (among other perioperative recommendations). These standardized, multidisciplinary recommendations are intended to improve decision-making and outcomes across cataract screening, surgery, and postoperative care in routine practice.

Wanten JC, Till V, Findl O et al. · Journal of cataract and refractive surgery · (2026) · View on PubMed ↗

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Cancer Immunotherapy Targets and Checkpoint/Cell Therapy Dynamics

Human monoclonal antibodies targeting α-Gal restrict IgE engagement of α-Gal syndrome allergens.

This study investigated human monoclonal antibodies (mAbs) targeting the α-Gal epitope (galactose-α-1,3-galactose) in individuals exposed to malaria, focusing on how these antibodies restrict IgE engagement in α-Gal syndrome. The authors isolated 42 α-Gal-specific mAbs that predominantly used the IGHV3 gene family and exhibited a range of somatic mutation frequencies, then screened them for reduced IgE binding to α-Gal syndrome allergens. These α-Gal–targeting IgE-blocking mAbs provide a mechanistic basis for developing more precise biologics for α-Gal syndrome.

Cho H, Seo Y, Sohn H et al. · The Journal of clinical investigation · (2026) · View on PubMed ↗

Multidisciplinary Delphi consensus on malignancy screening in patients with common variable immunodeficiency.

This multidisciplinary Delphi consensus studied standardized malignancy screening recommendations for adults with common variable immunodeficiency (CVID), a population with increased risk of hematologic malignancies and gastric carcinoma. The consensus highlighted substantial variability in current surveillance practices across centers and aimed to define evidence-based, practical screening intervals and modalities for CVID patients. Standardizing malignancy surveillance in CVID is clinically significant because it may reduce preventable morbidity and mortality by improving early detection in a high-risk immunodeficiency population.

Cabanero-Navalon MD, de Andrés-Martín A, Gil AA et al. · Frontiers in immunology · (2026) · View on PubMed ↗

Real-World Efficacy and Safety of Standard-of-Care Chimeric Antigen Receptor T-Cell (CART) and Bispecific T-Cell Engager (TCE) Therapies in Relapsed/Refractory Multiple Myeloma (RRMM).

This real-world study evaluated standard-of-care chimeric antigen receptor T-cell (CAR-T) versus bispecific T-cell engager (TCE) therapies in relapsed/refractory multiple myeloma (RRMM) using the US Flatiron Health Research Database (2021–2024). Among 419 patients (CAR-T n=220; TCE n=199), the study compared utilization, outcomes, and tolerability in routine practice, noting baseline differences such as younger age, better ECOG performance status, and higher prior autologous stem cell transplant rates in the CAR-T cohort. The findings are significant for clinicians because they contextualize effectiveness and safety of CAR-T and TCE approaches outside clinical trials, informing treatment selection in RRMM.

Theprungsirikul P, Wang R, Chang SH et al. · European journal of haematology · (2026) · View on PubMed ↗

Mitochondrial Complex I at the Crossroads of NK cell Dysfunction in Glioblastoma.

This mechanistic study examined how mitochondrial complex I activity, mediated by the gene NDUFA9, governs natural killer (NK) cell metabolic fitness and antitumor function in glioblastoma. The key finding was that impaired oxidative phosphorylation drives glutamine dependence, epigenetic repression of effector programs, and loss of NK cell activity, positioning mitochondrial fitness as an actionable vulnerability. This is scientifically significant because it identifies NDUFA9/complex I–linked metabolic control as a potential target to enhance NK-cell–based immunotherapy in solid tumors like glioblastoma.

Tiberti S, Rezvani K, Daher M · Cancer discovery · (2026) · View on PubMed ↗

Translational Challenges and Opportunities in mRNA Cancer Vaccines.

This mini-review studied translational challenges and opportunities for messenger RNA (mRNA) cancer vaccines, integrating clinical evidence from personalized platforms such as mRNA-4157 and BNT122 and off-the-shelf constructs including BNT111 and BNT113. The review highlighted key dualities—personalization versus shared antigen, potency versus safety, and speed versus durability—and proposed strategies such as modular vaccine design, prime-boost regimens, and adaptive trial approaches. The significance lies in guiding how next-generation mRNA vaccine platforms can be engineered and tested to improve scalability while maintaining durable antitumor immunity.

Jeong SD, Schrank BR, Mancuso JJ et al. · Cancer discovery · (2026) · View on PubMed ↗

GPRC5D-targeting bispecific and trispecific antibodies in multiple myeloma: Current evidence and emerging strategies.

This review studied GPRC5D-targeting T-cell–engaging bispecific and trispecific antibody strategies in relapsed/refractory multiple myeloma, focusing on agents beyond the currently approved talquetamab. The key finding was that multiple emerging constructs (including bispecific/trispecific antibodies and related approaches) are being developed to address clinical differences from BCMA and to overcome resistance mechanisms. The scientific and clinical significance is that it maps the evolving therapeutic landscape for GPRC5D, supporting rational combination and sequencing strategies to improve outcomes in RRMM.

Pan D, Kumar A, Lipof JJ et al. · Cancer · (2026) · View on PubMed ↗

A CAR-T Tonic Signaling Code Predicts Anti-Tumor Efficacy in Diffuse Midline Glioma.

This preclinical study examined whether a CAR-T “tonic signaling code” predicts anti-tumor efficacy in diffuse midline glioma using B7-H3 (MGA271-based) CAR-T constructs with different scFv designs. CAR variants with distinct tonic signaling profiles showed differences in persistence/exhaustion and anti-tumor activity, linking tonic signaling strength to therapeutic durability and outcomes. The work is significant for designing B7-H3 CAR-T therapies for pediatric diffuse midline glioma (DMG/DIPG) with improved persistence and more consistent efficacy.

Deng B, Zhong X, Xin D et al. · Neuro-oncology · (2026) · View on PubMed ↗

Precision nanomedicine for pulmonary diseases: from molecular targeting to clinical translation.

This review article surveyed how precision nanomedicine approaches for pulmonary diseases address limitations of conventional gene and drug therapies, focusing on molecular targeting and clinical translation. It highlights nanoparticle delivery strategies that improve therapeutic stability/bioavailability, enable controlled release, enhance cellular uptake and endosomal escape, and achieve tissue- and cell-specific targeting. The synthesis supports translational design principles for safer and more effective pulmonary therapeutics, including for infectious and genetic lung disorders.

Deng Z, Gao W, Do J et al. · Signal transduction and targeted therapy · (2026) · View on PubMed ↗

Integrated Immunotherapy Target Atlas for Ewing Sarcoma.

This study developed an integrated immunotherapy target atlas for Ewing sarcoma by converting the Deng et al. ESS32 32-gene signature into a practical 38-gene target set using tumor RNA expression plus normal-tissue and protein evidence. It identified and prioritized candidate antigens (including STEAP1, LINGO1, PRAME, CD99, CD276/B7-H3, and ENPP1) by integrating subcellular localization and therapeutic accessibility across 8 GEO datasets (n=854 samples). The atlas provides a rational, fusion-driven, low–tumor mutational burden antigen discovery framework to accelerate selection of immunotherapy targets for Ewing sarcoma.

Goldman C · Cancer genomics & proteomics · (2026) · View on PubMed ↗

Patterns of Response to Durvalumab Plus Tremelimumab and Atezolizumab Plus Bevacizumab in Patients With Unresectable Hepatocellular Carcinoma.

This retrospective single-center study analyzed response dynamics in 148 patients with unresectable hepatocellular carcinoma treated with durvalumab plus tremelimumab or atezolizumab plus bevacizumab, focusing on depth of response, time to response, and duration rather than only ORR/PFS/OS. It characterized how these immune checkpoint inhibitor combinations produce different response trajectories across patients. The findings are important for refining endpoint selection and improving interpretation of treatment benefit in unresectable HCC.

Kuwano A, Yada M, Tanaka K et al. · Anticancer research · (2026) · View on PubMed ↗

Engineering Lipid Nanoparticles through Integrated Compositional and Ligand Targeting Enhances β Cell-Directed RNA Delivery.

This study engineered lipid nanoparticles (LNPs) to enhance pancreatic β cell–directed RNA delivery by combining high-throughput compositional screening with surface conjugation of β cell–specific targeting ligands. It achieved a >148-fold increase in β cell transfection efficiency in vitro and >8-fold improvement in vivo (as reported in the abstract). The work is significant because it provides a scalable LNP design strategy to improve extrahepatic gene delivery to β cells, supporting development of β cell–targeted RNA therapeutics.

Yu D, Zhu Y, Roca-Rivada A et al. · ACS nano · (2026) · View on PubMed ↗

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Cancer Genomics/Epigenomics/Mechanisms of Resistance and Evolution

XY0206 targets FLT3-dependent resistance states in acute myeloid leukemia.

This study evaluated XY0206, a sunitinib-derived FLT3 inhibitor, in FLT3-dependent acute myeloid leukemia (AML) models and in settings of treatment-emergent, microenvironment-mediated resistance. XY0206 directly engaged FLT3 and suppressed STAT5, AKT, and ERK signaling, inducing apoptosis in FLT3-ITD AML cells while retaining antileukemic activity across multiple FLT3-dependent resistance models. Clinically, it supports XY0206 as a candidate next-generation FLT3-targeted therapy designed to overcome resistance to existing FLT3 inhibitors.

Shen L, Yang Y, Xu C et al. · The Journal of clinical investigation · (2026) · View on PubMed ↗

Comparative effectiveness of apalutamide-, abiraterone-, and enzalutamide-based doublets in mHSPC.

This study compared real-world effectiveness of androgen receptor pathway inhibitor doublets—apalutamide (APA), abiraterone (ABI), and enzalutamide (ENZA)—in metastatic hormone-sensitive prostate cancer (mHSPC) populations receiving ADT. Using a systematic review of observational/real-world studies (search through 22 Sep 2025), it assessed comparative outcomes across these APA+ADT, ABI+ADT, and ENZA+ADT regimens. The significance is to inform treatment selection in mHSPC where head-to-head randomized comparisons between these doublets are limited.

Valikovics AK, Bacsó D, Samaien N et al. · BJU international · (2026) · View on PubMed ↗

Tumor-Secreted ADAMTSL4 Activates Latent TGFβ1 to Drive Cancer Cachexia.

This study examined how tumor-secreted ADAMTSL4 drives cancer cachexia by activating latent TGFβ1 in preclinical models and in patients with colorectal and lung cancers. In mice, Adamtsl4 overexpression converted non-cachexia tumors into cachexia-inducing tumors, while Adamtsl4 deletion reduced muscle atrophy signatures and cachexia severity; mechanistically, ADAMTSL4 promoted local activation of TGFβ1 at muscle cell membranes by engaging the latency-associated peptide (LAP) of TGFβ1. The significance is identification of the ADAMTSL4–proTGFβ1 activation axis as a potential therapeutic target for cachexia.

Machado J, Karthikaisamy V, Mohr H et al. · Cancer discovery · (2026) · View on PubMed ↗

Metabolic Reprogramming of NSUN2-Mediated m5C Modification Promotes the Progression of Hepatocellular Carcinoma by Restricting Natural Killer Cell-Mediated Cytotoxicity.

This study examined how NSUN2-mediated m5C RNA methylation promotes hepatocellular carcinoma (HCC) progression by restricting natural killer (NK) cell–mediated cytotoxicity. Using a genome-wide CRISPR screen in HCC cells cocultured with NK cells and integrating downstream m5C-related profiling, the authors linked NSUN2 activity to metabolic reprogramming that suppresses NK cell function in the tumor microenvironment. The work identifies NSUN2/m5C-driven metabolic immunosuppression as a potential target to enhance NK-based anti-tumor immunity and overcome immunotherapy resistance.

Mao S, Fang Y, Gao J et al. · Cancer communications (London, England) · (2026) · View on PubMed ↗

Intratumoral Mycobacterium abscessus promotes cytidine deaminase mutagenesis in non-small cell lung cancer.

This study used integrated multi-omics in human non-small cell lung cancer (NSCLC) to determine whether intratumoral Mycobacterium abscessus drives tumor mutagenesis. It showed that bacterial nucleoside diphosphate kinase (NDK) phosphotransfers to IRF3 (non-canonical 1-phosphohistidine at H263), amplifying type I interferon signaling and sustaining APOBEC3A-associated cytidine deaminase mutagenesis. This mechanistic link between an intratumoral bacterium and APOBEC3A mutagenesis suggests microbiome-targeted strategies to reduce therapy-relevant tumor evolution.

Li X, Li MT, Ou KP et al. · Signal transduction and targeted therapy · (2026) · View on PubMed ↗

First-line anlotinib versus bevacizumab plus CapeOX in RAS/BRAF wild-type unresectable metastatic colorectal cancer (ANCHOR): a multicenter, prospective, randomized, phase 3 trial.

This multicenter, prospective, randomized phase 3 trial (ANCHOR; NCT04854668) compared first-line anlotinib versus bevacizumab plus CapeOX in RAS/BRAF wild-type unresectable metastatic colorectal cancer. The study randomized patients 1:1 to anlotinib (12 mg daily on days 1–14 of 21-day cycles) plus CapeOX or bevacizumab (7.5 mg/kg day 1) plus CapeOX, with maintenance using anlotinib or bevacizumab. If noninferiority is supported by outcomes, it would establish an oral multitargeted TKI strategy as an alternative to standard bevacizumab-based chemotherapy in this molecularly defined mCRC population.

Liu Y, Zhang Y, Lin R et al. · Signal transduction and targeted therapy · (2026) · View on PubMed ↗

Brassinin Inhibits Cell Survival and Autophagy via Modulation of ROS Production and the AMPKα-beclin1 Signaling Pathway in Gliomas.

This study tested the anticancer effects of brassinin, a Chinese cabbage-derived phytoalexin, in glioma cell lines and explored the underlying mechanism. It reported that brassinin inhibits cell survival and autophagy by modulating reactive oxygen species (ROS) production and the AMPKα–beclin1 signaling pathway, using assays including CCK8/colony formation, flow cytometry/Western blotting for apoptosis and cell cycle, and LC3/p62 analyses. These mechanistic data support brassinin as a potential therapeutic candidate targeting ROS-AMPKα-beclin1 control of autophagy in gliomas.

Rauniyar S, Wang Y, Zhang Q et al. · Phytotherapy research : PTR · (2026) · View on PubMed ↗

Factors associated with the effectiveness and safety of Janus kinase inhibitors in rheumatoid arthritis: an 8-year observational study from the JAGUAR Registry.

This 8-year retrospective observational analysis used the multicentre JAGUAR Registry to study factors associated with effectiveness and safety of Janus kinase inhibitors in rheumatoid arthritis patients, using treatment retention and discontinuation reasons (lack of effectiveness vs adverse drug reactions). It evaluated retention with Cox regression and Kaplan–Meier curves across 435 JAK inhibitor treatment episodes at 1 and 4 years. The findings aim to identify patient- and treatment-related predictors of sustained benefit and tolerability to guide real-world JAK inhibitor selection in RA.

Martinez-Molina C, Frade-Sosa B, Diaz-Torné C et al. · RMD open · (2026) · View on PubMed ↗

Synergy of KRAS-inhibitor Daraxonrasib Combined With Recombinant Methioninase on Pancreatic-cancer Cells But Not Normal Cells.

This in vitro study tested whether the KRAS inhibitor daraxonrasib synergizes with recombinant methioninase (rMETase) in pancreatic cancer cells harboring the KRAS G12D mutation versus normal fibroblasts. It found synergy on KRAS G12D pancreatic cancer cells while sparing normal cells, using cell viability assays (e.g., WST-based measurement). The work is significant as it supports a targeted combination strategy that may improve anti-tumor efficacy while potentially reducing toxicity.

Kim J, Han Q, Li S et al. · Anticancer research · (2026) · View on PubMed ↗

Predicting cellular responses to perturbation across diverse contexts with State.

This study developed and evaluated State, a machine-learning model trained on single-cell gene expression data to predict transcriptomic responses to perturbations across heterogeneous cellular contexts. State improved discrimination of perturbation effects on large datasets by >30% and more accurately identified differentially expressed genes across genetic, signaling, and chemical perturbations than baseline approaches. The work provides a more generalizable framework for forecasting cellular outcomes of perturbations, supporting more reliable interpretation of single-cell perturbation experiments.

Adduri AK, Gautam D, Bevilacqua B et al. · Cell · (2026) · View on PubMed ↗

An IRAK1-snRNA axis activates ATM to promote accurate repair within transcriptionally active chromatin.

This study investigated how an IRAK1–small nuclear RNA (snRNA) axis regulates ATM activation to promote accurate homologous recombination (HR) repair within transcriptionally active chromatin in human cancer cells. IRAK1 phosphorylated spliceosomal SR-rich proteins to recruit snRNA to DNA double-strand break (DSB) sites, which condensed the MRN (MRE11–RAD50–NBS1) complex near active chromatin to form an ATM activation platform and enhance accurate repair. These results define a mechanistic link between splicing machinery and DNA repair fidelity, suggesting new targets to improve genome stability in cancer.

Nie C, Liu W, Zhang L et al. · Molecular cell · (2026) · View on PubMed ↗

Promiscuous RNA binding by WDR5 remodels the KMT2A (MLL1) histone methyltransferase complex to an inactive state.

This study examined RNA binding properties of WDR5, an essential subunit of the KMT2A/MLL1 histone methyltransferase complex, and how this affects the complex’s chromatin state. WDR5 bound RNA promiscuously in an abundance- and length-dependent manner (rather than sequence motif–dependent recruitment), with RNA binding involving multiple surfaces overlapping interfaces with MLL1 complex subunits, and this remodeled the KMT2A complex into an inactive state. The findings revise models of RNA–chromatin factor specificity and suggest that cellular RNA abundance can directly tune epigenetic activity via WDR5.

Kainth AS, Sirjoosingh P, Werner MS et al. · Molecular cell · (2026) · View on PubMed ↗

Lysyl oxidase inhibition disrupts mitochondrial homeostasis to create vulnerability to ferroptosis in TNBC.

This study tested whether lysyl oxidase (LOX) inhibition alters mitochondrial homeostasis to create ferroptosis vulnerability in triple-negative breast cancer (TNBC). Inhibiting LOX disrupted mitochondrial homeostasis and sensitized TNBC to ferroptosis, mediated by LOX interaction with PARKIN and oxidation of PINK1, which suppressed PARKIN phosphorylation, stabilized HIF-1α, and increased glycolysis. The work identifies LOX as a metabolic/mitochondrial regulator that can be therapeutically targeted to induce ferroptosis in TNBC.

Saatci O, Ulukan B, Cetin M et al. · Cell reports. Medicine · (2026) · View on PubMed ↗

Non-genetic remodeling drives leukemia propagation and reveals actionable vulnerabilities in acute myeloid leukemia.

This study analyzed how non-genetic remodeling drives leukemia propagation in acute myeloid leukemia (AML) using serial patient-derived xenotransplantation and integrated single-cell transcriptomics with multi-omics profiling. The longitudinal model showed a predominantly non-genetic trajectory with leukemia-initiating capacity increasing over time, following a conserved stage-specific pattern across epigenetic, transcriptional, and proteomic layers, supported by ribosome profiling and rRNA 2′-O-methylation analyses. These results reveal actionable vulnerabilities tied to disease evolution that could inform stage-matched therapeutic strategies in AML.

Larrue C, Angelino P, Mouche S et al. · Cell reports. Medicine · (2026) · View on PubMed ↗

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Metabolic Disease and Endocrinology (including Obesity/Diabetes)

Gut microbiome-derived metabolites as prognostic biomarkers in heart failure: a systematic review and meta-analysis.

This systematic review and meta-analysis synthesized evidence (through February 2026) on whether gut microbiome-derived metabolites are prognostic biomarkers for heart failure outcomes, focusing on all-cause mortality and major adverse cardiac events (MACE). Across included studies, specific gut microbial metabolites showed statistically significant associations with HF prognosis using random-effects pooling of hazard ratios, with subgroup analyses by HF phenotype and etiology. These findings suggest metabolite signatures could help risk-stratify HF patients and guide future biomarker validation studies.

Koeckerling D, Reddy RK, Guivala SJ et al. · European journal of heart failure · (2026) · View on PubMed ↗

Associations Between Glucagon-Like Peptide-1 Receptor Agonists (GLP-1RAs) and Cancer Risk: A Systematic Review and Meta-Analysis.

This systematic review and meta-analysis evaluated whether glucagon-like peptide-1 receptor agonists (GLP-1RAs) used in adults for any indication are associated with cancer incidence, using literature searches in MEDLINE and EMBASE up to September 17, 2025. The pooled evidence did not show a clear increase in cancer risk with GLP-1RA use (with effect estimates summarized across included studies). Clinically, this informs ongoing safety surveillance and helps contextualize cancer-risk concerns as GLP-1RAs expand for type 2 diabetes and cardiovascular risk reduction.

Lalani I, Nambayan R, Carbonell C et al. · Cancer control : journal of the Moffitt Cancer Center · (2026) · View on PubMed ↗

Efficacy of SGLT2 inhibitors, GLP-1 receptor agonists, and aerobic exercise for moderate-to-severe obstructive sleep apnea in overweight or obese patients: a network meta-analysis.

This network meta-analysis compared the efficacy of SGLT2 inhibitors, GLP-1 receptor agonists, and aerobic exercise for improving obstructive sleep apnea (OSA) severity in overweight or obese patients. By synthesizing direct and indirect evidence across included studies, it estimated relative effects of each intervention on OSA outcomes (e.g., severity measures) in this population. Clinically, the results help rank treatment options for obesity-associated OSA when head-to-head trials are limited.

Zheng J, Zhu Z, Bao Y et al. · Frontiers in endocrinology · (2026) · View on PubMed ↗

The Efficacy of Exercise Therapy for Nonspecific Chronic Low Back Pain According to the FITT Principle: A Systematic Review With Meta-analyses.

This systematic review with meta-analyses studied exercise therapy interventions for adults with nonspecific chronic low back pain (>3 months), comparing trials that differed in at least one FITT component (frequency, intensity, type, time). The key finding was that the efficacy of exercise therapy on disability could be evaluated by analyzing effects across specific FITT components using random-effects meta-analyses. The clinical significance is that it helps clinicians tailor exercise prescriptions more precisely by identifying which FITT elements are most supported for improving disability in chronic nonspecific low back pain.

Blanco-Heras L, Rebollo-Salas M, Cardellat-González M et al. · The Journal of orthopaedic and sports physical therapy · (2026) · View on PubMed ↗

Real-World Evidence on the Effectiveness and Safety of Vosoritide in Latin American Patients With Achondroplasia (EVOLAC).

This retrospective, multicenter, multinational real-world cohort study evaluated effectiveness, safety, treatment continuity, and caregiver-reported outcomes of vosoritide (a C-type natriuretic peptide analog) in children with molecularly confirmed achondroplasia treated in Latin American centers (Argentina, Colombia, and Uruguay). Vosoritide use in routine practice showed clinically meaningful effectiveness with an acceptable safety profile and supported ongoing treatment across heterogeneous health systems. These findings extend clinical-trial evidence for FGFR3 gain-of-function achondroplasia to Latin American real-world settings, informing implementation and monitoring of vosoritide in diverse care environments.

De Victor J, Gil ED, Pabletich F et al. · American journal of medical genetics. Part A · (2026) · View on PubMed ↗

Integrative Analysis Uncover the Effects and Multi-Omics Features of Thigh Muscle Fat Infiltration.

This integrative analysis studied thigh muscle fat infiltration (TMFI) measured by magnetic resonance imaging in 55,120 UK Biobank participants to determine health impacts and underlying multi-omics correlates. Higher TMFI was associated with increased all-cause mortality and major system-specific diseases, and TMFI mediated effects of lifestyle factors; a GWAS identified 79 lead SNPs linked to TMFI and enabled polygenic risk scoring. These findings are clinically relevant because they connect MRI-defined muscle fat infiltration to genetic susceptibility and downstream disease risk, supporting TMFI as a potential biomarker and target for intervention.

Zhang Y, Dang Q, Zong X et al. · Aging cell · (2026) · View on PubMed ↗

Association of tirzepatide with changes in OSA-related measures based on baseline characteristics - post hoc analyses of SURMOUNT-OSA.

These post hoc analyses studied associations between tirzepatide (a GIP/GLP-1 receptor agonist; 10 mg or 15 mg) and changes in obstructive sleep apnea (OSA) measures—apnea–hypopnea index (AHI), hypoxic burden, body weight, and blood pressure—stratified by baseline characteristics in adults with moderate-to-severe OSA from the SURMOUNT-OSA Phase 3 trials. Tirzepatide was associated with improvements in OSA-related outcomes, with effect sizes varying descriptively by baseline factors such as BMI, neck circumference, age, sex, and baseline AHI. The findings are significant for personalizing anti-obesity pharmacotherapy to reduce OSA severity and related cardiovascular risk.

Falcon B, Xie CC, Redline S et al. · Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine · (2026) · View on PubMed ↗

Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.

This phase 3, double-blind, randomized, placebo-controlled trial evaluated DTX401, an AAV8 vector delivering the human G6PC gene, in patients ≥8 years with glycogen storage disease type Ia (GSDIa) due to biallelic G6PC variants. The primary endpoint compared percent change from baseline to week 48 in daily cornstarch intake between DTX401 and placebo groups, with participants randomized 1:1 and a crossover after the primary efficacy period. The results are intended to establish clinical efficacy and inform regulatory-grade evidence for AAV gene therapy in a rare, life-threatening metabolic disorder.

Mitchell JJ, Abdenur JE, de Boer F et al. · Journal of inherited metabolic disease · (2026) · View on PubMed ↗

Dendrobine ameliorates diabetic cardiomyopathy by targeting KEAP1 to disrupt KEAP1-NRF2 interaction and activate antioxidant defense.

This study evaluated dendrobine, an active alkaloid from Dendrobium officinale, for treatment of diabetic cardiomyopathy (DCM) and tested whether it targets the KEAP1–NRF2 antioxidant pathway. In db/db mice and high-glucose–challenged in vitro models, dendrobine disrupted the KEAP1–NRF2 interaction by targeting KEAP1, thereby activating NRF2-dependent antioxidant defenses and ameliorating DCM phenotypes. The findings support dendrobine as a mechanistically defined candidate for boosting antioxidant signaling in diabetic heart disease.

Xiang X, Lin X, Wu Q et al. · Phytomedicine : international journal of phytotherapy and phytopharmacology · (2026) · View on PubMed ↗

An integrated reference atlas of human skeletal muscle.

This study constructed an integrated reference atlas of human skeletal muscle by harmonizing multiple adult human scRNA-seq and snRNA-seq datasets and benchmarking integration strategies. The authors generated a unified atlas of ~122,000 cells with modality-aware marker panels and validated selected findings by immunofluorescence in muscle biopsies. This resource improves reproducibility and cross-study comparability in skeletal muscle single-cell research, enabling more consistent cell-type annotation and downstream analyses.

Nelke C, Babilon F, Schroeter CB et al. · EBioMedicine · (2026) · View on PubMed ↗

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Renal Disease and Kidney Injury/Biomarkers

The SLC15A4-TASL complex is essential for lupus development in mice.

This study examined the role of the endolysosomal SLC15A4–TASL signaling axis in mouse models of systemic lupus erythematosus (SLE), focusing on downstream IRF5 activation. Genetic ablation of Tasl (TaslKO) and its paralogue Tasl2 (TaslDKO) ameliorated or fully prevented autoimmune manifestations driven by Faslpr loss of function, indicating broad requirement for SLC15A4–TASL in lupus pathogenesis across three genetic SLE models. These findings identify the SLC15A4–TASL–IRF5 pathway as a mechanistic vulnerability for therapeutic targeting in SLE.

Drobek A, Delacrétaz M, Vasilakou A et al. · Proceedings of the National Academy of Sciences of the United States of America · (2026) · View on PubMed ↗

Mechanosensitive phosphorylation of NFATC4 at S213/S217 drives fibroblast-to-myofibroblast transition and fibrosis.

This mechanistic study examined how mechanosensitive phosphorylation of the transcription factor NFATC4 at serines 213/217 regulates fibroblast-to-myofibroblast transition in primary human lung fibroblasts. Using phosphoproteomics and stiffness-controlled culture on fibronectin-coated substrates, the authors identified a stiffness threshold (2–8 kPa) above which cells transitioned to a CTHRC1+/ACTA2+ myofibroblast state with stiffness-dependent NFATC4 S213/S217 phosphorylation. The results link mechanical cues to NFATC4 activation, highlighting a potential target pathway to modulate fibrosis progression.

Kadri S, Mattner LF, Zeng Z et al. · The Journal of clinical investigation · (2026) · View on PubMed ↗

PUS7-Mediated Pseudouridylation of TGFBI Drives Vascular Remodeling in Pulmonary Hypertension.

This study investigated the role of pseudouridine synthase 7 (PUS7) in pulmonary hypertension by mapping RNA pseudouridylation and testing how PUS7 drives vascular remodeling. Using bisulfite-induced deletion sequencing to generate a single-base resolution pseudouridine (Ψ) landscape in lung tissues from patients with pulmonary hypertension, the authors assessed PUS7 expression in hypoxic pulmonary artery endothelial cells and implicated PUS7-dependent pseudouridylation in remodeling processes. Scientifically, it identifies PUS7 as a previously uncharacterized RNA-epigenetic regulator in PH, suggesting a new therapeutic axis for vascular remodeling.

Zhang J, Li Y, He M et al. · Circulation · (2026) · View on PubMed ↗

LDLR Variant Classification Through Activity-Normalized Prime Editing Screening.

This study developed an activity-normalized prime editing screening pipeline to classify LDLR variants by measuring their functional impact on LDL-cholesterol (LDL-C) uptake. Using prime editing guide RNAs to introduce 5184 LDLR coding variants and quantifying LDL-C uptake in a functional assay, the authors derived activity-normalized effects to support variant pathogenicity classification. This provides a scalable, experimentally grounded approach to reclassify uncertain LDLR variants and enable earlier, more accurate lipid-lowering and cascade testing.

Zhou PJ, Velimirovic M, Yu T et al. · Circulation · (2026) · View on PubMed ↗

Long-acting growth hormone: An updated Growth Hormone Research Society consensus statement.

This updated Growth Hormone Research Society consensus statement studied the evidence base and clinical recommendations for long-acting growth hormone (LAGH) preparations in pediatric growth hormone deficiency (GHD) and selected non-GHD short stature indications, and in some regions adult GHD. The consensus concluded that, across available LAGH products, short- to mid-term efficacy appears comparable to daily somatropin when dosed appropriately, with data extending up to ~7 years in more than 8,000 individuals. The update is clinically significant because it refines guidance for routine LAGH use and supports evidence-informed selection and monitoring of patients transitioning from daily GH.

Schilbach K, Clayton P, Agrawal N et al. · European journal of endocrinology · (2026) · View on PubMed ↗

Expert Consensus on the Diagnosis and Monitoring of Paroxysmal Nocturnal Hemoglobinuria (PNH): An Algorithmic Approach in an Era of New Treatments.

This Italian expert consensus article studied current challenges in diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria (PNH) and proposed an algorithmic approach in the era of complement inhibitors. The consensus addressed which patients should be screened, which laboratory tests to use, and how to structure initial work-up and longitudinal monitoring. This standardization is clinically significant because it aims to improve timely diagnosis and optimize complement-inhibitor management in a rare disease with evolving treatment paradigms.

Fattizzo B, Marchetti M, Cannizzo E et al. · European journal of haematology · (2026) · View on PubMed ↗

Genome-wide DNA methylation analysis revealed epigenetic mechanism underlying end-stage renal disease.

This study performed a large-scale two-stage epigenome-wide association study of end-stage renal disease (ESRD) using DNA methylation profiling in 704 controls and 1031 ESRD cases. It identified 52 ESRD-associated differentially methylated CpG sites consistently replicated across diverse primary kidney diseases, implicating 144 candidate genes enriched in inflammatory and immune pathways. These shared methylation signatures provide candidate biomarkers and mechanistic targets for ESRD progression beyond the initiating kidney disorder.

Zhou X, Shi D, Xu J et al. · Nature communications · (2026) · View on PubMed ↗

Nintedanib-Associated Glomerular Endothelial Injury With Secondary Collapsing Focal Segmental Glomerulosclerosis: Potential Role of Sodium-Glucose Cotransporter 2 Inhibition.

This case report studied a 69-year-old man with idiopathic pulmonary fibrosis who developed nintedanib-associated nephrotic syndrome and renal dysfunction with glomerular injury consistent with secondary collapsing focal segmental glomerulosclerosis. It described the clinical course occurring 16 months after starting nintedanib and explored a potential management role for sodium-glucose cotransporter 2 (SGLT2) inhibition. The report is clinically significant because it highlights a rare but serious nintedanib nephrotoxicity phenotype and suggests a possible therapeutic strategy when glomerular injury occurs.

Furuto Y, Hashimoto H, Yoshino D et al. · Nephrology (Carlton, Vic.) · (2026) · View on PubMed ↗

Cardiovascular Outcomes and Acute Human Metapneumovirus Infection in Adults: A Systematic Literature Review.

This PRISMA-guided systematic literature review studied adults with laboratory-confirmed acute human metapneumovirus (HMPV) infection to quantify the prevalence of pre-existing cardiovascular disease and the incidence of acute cardiovascular events. It screened MEDLINE, Embase, and Google Scholar (Jan 2000–Sept 2025) and included 18 observational studies and clinical trials reporting cardiovascular outcomes in adults ≥18 years. The review is significant because it clarifies the cardiovascular risk profile of HMPV, informing clinicians and researchers about potential acute cardiovascular complications beyond respiratory effects.

Loubet P, Roubille F, Launay O et al. · The Journal of infection · (2026) · View on PubMed ↗

Genomic and transcriptomic features of HBV integration in treatment-naïve, HBeAg-positive children with chronic HBV infection.

This study characterized genomic and transcriptomic features of hepatitis B virus (HBV) integration and local immune responses in treatment-naïve, HBeAg-positive children with chronic HBV infection. Using probe-based capture genomic analyses in 18 children and 28 adults, and spatial transcriptomics in 12 children (plus 3 adults from a public dataset), the study found age-associated differences in HBV integration landscape and associated transcriptional/immune responses. These data define how HBV integration behaves in pediatric infection and can inform age-tailored strategies for curing chronic HBV.

Wu N, Han Y, Tang J et al. · EBioMedicine · (2026) · View on PubMed ↗

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Neurology and Neuropsychiatric Disorders (Biomarkers, Genetics, Circuitry)

Musculoskeletal Treatment Mechanisms: A Puzzle That May Never Be Fully Solved.

This commentary studied the difficulty of explaining mechanisms behind musculoskeletal treatments, focusing on why exercises, manual techniques, and psychologically informed interventions often help despite limited evidence for the intuitive mechanisms clinicians propose. The key finding was that mechanistic research frequently isolates single pathways under controlled conditions that do not capture the multifactorial, interacting physiological, psychological, and social processes seen in real-world recovery. The significance is that it reframes expectations for mechanism discovery and supports more integrative approaches to understanding and optimizing musculoskeletal care.

Cook CE, Keter DL · The Journal of orthopaedic and sports physical therapy · (2026) · View on PubMed ↗

The WNT/β-catenin Pathway and Matrix Metalloproteinase-9 in hypermobile Ehlers-Danlos Syndrome and Autism Spectrum Disorder: A Possible Connection.

This article studied a potential biological connection between the WNT/β-catenin pathway and matrix metalloproteinase-9 (MMP-9) in hypermobile Ehlers-Danlos syndrome (hEDS) and autism spectrum disorder (ASD). The key finding was the proposed mechanistic link based on overlapping comorbidity patterns and the involvement of WNT/β-catenin signaling and MMP-9 in tissue/connective and neurodevelopmental processes. The significance is that it generates testable hypotheses for shared pathways that could inform future biomarker research and targeted interventions across hEDS and ASD.

Douglas AC, Staas DJ · Journal of musculoskeletal & neuronal interactions · (2026) · View on PubMed ↗

Increased Libido Following Brivaracetam Initiation: A Case Report of Temporal Lobe Epilepsy.

This case report described a woman in her 70s with drug-resistant temporal lobe epilepsy who developed increased libido after initiating brivaracetam (BRV). The key observation was a temporal association between BRV initiation and onset of hypersexual symptoms, in a context where sexual dysfunction is often underassessed in epilepsy care. Clinically, it suggests BRV may be associated with changes in sexual behavior in some patients, warranting monitoring and further study of ASM-related sexual side effects.

Horinouchi T, Matsuyama T, Mito M et al. · Neuropsychopharmacology reports · (2026) · View on PubMed ↗

Dlg5 and Cadherins cooperate to ensure peripheral glia and septate junction integrity.

This Drosophila RNAi screen studied which PDZ-domain proteins regulate peripheral glia sheath formation and septate junction integrity during larval development. It identified Dlg5 as a key scaffolding protein cooperating with cadherins to maintain peripheral glia morphology and septate junction integrity. These findings define a conserved adhesion/scaffold pathway controlling non-myelinating glial organization, informing how junctional architecture supports peripheral nerve function.

Das M, Cheng D, Matzat T et al. · The Journal of neuroscience : the official journal of the Society for Neuroscience · (2026) · View on PubMed ↗

Glia-Derived Upd1 Promotes Dendritic Remodeling via Neuronal JAK/STAT-CK2α Signaling.

This study investigated whether glia non-cell-autonomously regulate dendritic pruning in Drosophila class IV dendritic arborization (C4da) sensory neurons. It found that glia-derived Unpaired1 (Upd1) promotes dendritic remodeling by activating neuronal JAK/STAT signaling and downstream CK2α (CK2α) signaling during development in both male and female flies. The work clarifies a specific glia-to-neuron signaling axis that controls pruning, offering a mechanistic framework for understanding circuit refinement.

Huang E, Yuan Y, Wang S et al. · The Journal of neuroscience : the official journal of the Society for Neuroscience · (2026) · View on PubMed ↗

Neurofibromatosis Type I: Consensus on Current Terminology for Neurofibromas, as a Basis for Recommendations on the Use of MEK Inhibitors.

This multidisciplinary consensus paper studied and harmonized terminology for neurofibromatosis type 1 (NF1) neurofibromas, especially plexiform neurofibromas, to support consistent clinical and research communication. It proposed standardized definitions and usage of current terms to reduce ambiguity across pathology, radiology, and clinical practice, explicitly linking terminology to recommendations for MEK inhibitor use. The consensus is significant because consistent classification improves diagnosis, risk stratification, and appropriate selection of MEK inhibitor therapy in NF1.

Baldino L, Koeppen JA, Kammermeier J et al. · Anticancer research · (2026) · View on PubMed ↗

Endogenous opioid dynamics in the dorsal striatum sculpt neural activity to promote goal-directed action.

This study examined endogenous dynorphin–kappa opioid receptor (KOR) signaling in dorsomedial striatum medium spiny neurons (MSNs) and its role in goal-directed behavior. Local, time-locked dynorphin release from dorsomedial striatum MSNs was necessary and sufficient to promote goal-directed action. These findings establish a spatiotemporally precise mechanism for endogenous opioid control of striatal circuits, highlighting dynorphin/KOR signaling as a potential target for disorders of goal-directed behavior.

Gowrishankar R, Hjort MM, Elerding AJ et al. · Cell · (2026) · View on PubMed ↗

Plasma neurofilament light for early differentiation of multiple system atrophy from Parkinson disease.

This study assessed whether plasma neurofilament light chain (NfL) can differentiate multiple system atrophy (MSA) from Parkinson disease (PD) early, and whether adding glial fibrillary acidic protein (GFAP), total tau (t-tau), and p-tau217 improves performance. In a multicenter setting, plasma NfL showed diagnostic utility for early MSA–PD differentiation within about two years of symptom onset, and the study evaluated incremental biomarker value from GFAP and tau measures. The results support a clinically practical blood-based biomarker approach to reduce diagnostic uncertainty and improve prognosis, referral, and trial enrollment.

Yu Z, Zheng Y, Kou W et al. · EBioMedicine · (2026) · View on PubMed ↗

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Generated automatically on September 02, 2026 from PubMed’s trending articles. Summaries are AI-generated; always consult the original publication for clinical or research decisions.