2026년 4월 11일 Pubmed Today

Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 with transfusion-dependent β-thalassemia (TDT). Standard of care (SOC) for TDT includes regular red blood cell transfusions (RBCTs) and iro…

Desmoplastic small round cell tumor (DSRCT) is an ultra-rare sarcoma with limited treatment options. Here, we show that comprehensive molecular profiling informs diagnosis and individualized therapy in this disease. We report the results of whole-genome/exome, transcriptome, and DNA methylome analys…

Second primary cancers (SPCs) pose an increasingly significant clinical challenge for survivors of hematologic malignancies, attributed to improved therapeutic outcomes and prolonged survival. The development of SPCs is influenced by a complex interplay of treatment-related factors, genetic suscepti…

Integrating spatial omics with artificial intelligence is likely to advance biomarker research and diagnostics, with the potential to pair mechanistic insight into spatial target biology. By developing scalable, reproducible quantification in routine pathology, it can help bridge discovery, validati…

Activation of the STING pathway represents a potent strategy for cancer immunotherapy. However, the instability and systemic toxicity of STING agonists, especially cyclic dinucleotides (CDNs), limit their use via local administration with poor efficacy against metastatic or inaccessible tumors. Here…

Immunotherapies such as checkpoint inhibitors (i.e. anti-PD-1) and peptide-based therapies (DPX-Survivac) have strong potential for treatment of epithelial ovarian cancer, the most lethal gynecological malignancy. Magnetic resonance imaging (MRI) can be used to track tumor growth and iron-labeled im…

Chimeric antigen receptor macrophages (CAR-M) therapy holds significant clinical potential, while its application is severely compromised by poor in vivo colonization and limited persistence. Here, we report a 3D-printed implantable CAR-M (iCAR-M) system, composed of a gelatin methacrylate (GelMA) s…

Natural killer (NK) cells are key effectors in antitumor immunity, yet their function is markedly suppressed by transforming growth factor-β (TGF-β) in the tumor microenvironment. SMAD7 is an established intracellular antagonist of TGF-β signaling, but its specific role within NK c…

Retinal involvement in autoimmune polyendocrine syndrome type 1 (APS1), a rare monogenic autoimmune disorder caused by mutations in the AIRE gene, is increasingly recognised but remains poorly defined. Prior reports suggest a variable phenotype, ranging from mild changes to severe vision loss, often…

Recent innovations in melanoma treatment with immune checkpoint blockade (ICB) have improved overall outcomes for patients, however over 50% of patients still develop resistance to treatment. These patients either have intrinsic resistance, and never respond to therapy, or develop acquired resistanc…

To develop Colombian, evidence-informed consensus recommendations for perioperative systemic therapy and unresectable stage III non-small cell lung cancer (NSCLC), considering local testing and access constraints….

Tumor-associated macrophages (TAMs) are central regulators of the tumor microenvironment (TME), with their metabolic states critically influencing tumor progression or regression. Although reprogramming TAM metabolism is a promising therapeutic avenue, clinical translation remains challenging due to…

Patients with newly diagnosed multiple myeloma (NDMM) who are ineligible for or not proceeding to autologous stem-cell transplantation (ASCT)-often because of age or frailty-have limited opportunities to receive multiple effective lines of therapy, underscoring the need for novel frontline strategie…

The efficacy of nanocatalytic therapy is constrained by the limited availability of endogenous hydrogen peroxide (H2O2) as a reaction substrate, finite catalytic activity of nanozymes and rapid scavenging by intracellular antioxidants, hindering their accumulation at target sites to therapeutic conc…

Sorafenib is the first-line therapy for advanced hepatocellular carcinoma (HCC). However, acquired resistance to sorafenib remains a significant challenge. Previous studies have shown that sorafenib treatment induces the formation of truncated O-glycans in HCC cells, but the relationship between sor…

Thymic epithelial tumors (TETs), comprising thymomas, thymic carcinomas, and thymic neuroendocrine neoplasms, are rare prevascular (anterior) mediastinal malignancies characterized by histologic and clinical heterogeneity. Their management is complex because of overlapping pathologic features, varia…

Immunotherapy has become a mainstream part of cancer care, but the immune system is also critical for wound healing. Dysregulating immunity to treat cancer raises concerns about potential iatrogenic effects on postoperative recovery. Despite its growing use, immunotherapy’s impact on surgical wound …

The ASCO 2025 meeting featured several phase III trials with the potential to impact gastrointestinal cancer management. CheckMate 577 reported mature overall survival (OS) data for adjuvant nivolumab in esophageal/GEJ cancer; however, OS benefit was not statistically significant in the intent-to-tr…

Cervical cancer remains a major global health challenge, particularly in advanced stages where prognosis is poor despite recent therapeutic advances. The immunological landscape of cervical cancer offers a rationale for strategies aimed at enhancing tumor-specific immune responses. Based on this rat…

Lung cancer is the most frequent cause of cancer-related mortality globally. In recent years, the treatment landscape for advanced-stage non-small-cell lung cancer (NSCLC) has been transformed by the advent of immunotherapy, which has yielded unprecedented and durable clinical responses for some pat…

The existing TNM staging system provides insufficient prognostic information in gastric cancer (GC) patients. This study aims to establish a pathomics signature of GC (PSGC) that uses deep learning (DL) to directly analyze H&E slides for predicting GC outcomes. We propose a multi-scale graph neu…

Developing resistance to chemotherapy drugs and evading the killing effect of the immune system are the main obstacles in the clinical treatment of gastric cancer. However, the potential mechanism remains poorly understood. N-acetyltransferase 10 (NAT10) catalyzes the N4-acetylcytidine (ac4C) modifi…

Personalized immunotherapy strategies are urgently needed for patients with locoregionally advanced nasopharyngeal carcinoma (NPC). We aim to identify biomarkers predictive of immunotherapy benefits, using data from the phase III CONTINUUM (ClinicalTrials.gov identifier: NCT03700476) and DIPPER (Cli…

While immunotherapy is a promising treatment strategy for cancer, the majority of head and neck squamous cell carcinoma (HNSCC) patients treated with single-agent immunotherapy do not respond. Therefore, researchers are investigating combination treatments with immunostimulatory molecules that can m…

The stimulator of interferon genes (STING) has emerged as a promising target for cancer immunotherapy. However, STING agonists alone have demonstrated limited clinical success. Indoleamine 2,3-dioxygenase 1 (IDO1) has been implicated as a key resistance mechanism that undermines the antitumor effect…

Breast cancer remains a leading cause of cancer-related mortality in women, with the clinical efficacy of immune-checkpoint inhibitors limited by low tumor immunogenicity. Intracellular copper accumulation can induce cuproptosis, an immunogenic form of cell death that enhances antitumor immunity; ho…

Cancer and depression exhibit a clinically significant bidirectional association, as evidenced by the key findings: cancer patients are prone to depression, and depressed patients are prone to cancer. This may help explain why depression is commonly observed in cancer populations, where it is associ…

Conventional transarterial chemoembolization (TACE) regimens for hepatocellular carcinoma (HCC) are often compromised in efficacy due to hypoxia and acidosis within the tumor microenvironment (TME), frequently leading to unsatisfactory treatment outcomes and tumor recurrence. To overcome these limit…

Activation of cyclic guanosine monophosphate-adenosine monophosphate synthase (cGAS)-interferon gene stimulator (STING) pathway has demonstrated significant potential in cancer treatment due to its crucial role in bridging the innate and adaptive immunity. However, clinical attempts of current cGAS-…

Pharmacological targeting of ATR (ataxia telangiectasia and Rad3-related kinase), the master regulator of replication stress response, is emerging as a promising anticancer strategy. Despite the documented immune-modulatory effects of ATR inhibitors (ATRi), the immune evasion mechanisms constraining…

The efficacy of immune checkpoint blockade (ICB) against glioblastoma (GBM) is significantly constrained by the blood-brain barrier (BBB) and the immunosuppressive tumor microenvironment associated with glioma stem cells (GSCs). To address these challenges, anti-programmed death-ligand 1 antibodies …

Melittin, the principal bioactive component of bee venom, exhibits potent anticancer activity. However, its clinical application is hindered by nonspecific hemolytic toxicity. In this study, we developed a pH-responsive polymeric-peptide complex (PCM) to improve anti-tumor activity of melittin while…

This randomized phase I/II clinical trial (NCT03617328) was designed to test whether administration of systemic agonistic anti-CD27 antibody (varlilumab) concurrent with a melanoma vaccine is safe and enhances vaccine immunogenicity….

Despite extensive clinical endeavors to enhance high-grade serous ovarian cancer (HGSOC) detection and treatment, an alarming half of diagnosed women succumb annually to this disease. Significantly, nearly all HGSOC cases manifest ascites at diagnosis, a poor prognostic indicator. Malignant ascites …

There is a growing body of evidence supporting neoadjuvant systemic therapy to improve outcomes for people with early-stage non-small cell lung cancer (NSCLC). However, not all patients will respond, necessitating the need to explore novel combinations and predictors of response to neoadjuvant thera…

Somatic variant calling algorithms typically detect mutations in cancer genomes by comparing sequence data from a tumor sample against a matched normal sample. However, matched normal samples are often unavailable in clinical diagnostics or retrospective analyses of archival tumor samples in biobank…

Tumors foster an immunosuppressive microenvironment to evade the antitumor immune response. However, the influence of intratumoral immunosuppressive steroids on tumor-infiltrating natural killer (NK) cells and their implications for effective immunotherapy has remained largely unexplored. Here, we r…

ABBV-368 is a humanized monoclonal antibody that targets the costimulatory receptor OX40. Here, we investigate a treatment strategy with ABBV-368 combined with the investigational toll-like receptor 9 agonist tilsotolimod, the programmed cell death 1 inhibitor budigalimab, and nab-paclitaxel in pati…

Checkpoint blockade therapies have demonstrated clinical benefit across multiple cancer types; however, many patients with immune cell infiltration remain non-responsive or develop resistance. This suggests that additional, unidentified regulatory pathways limit treatment efficacy. The purpose of th…

Autologous tumor-infiltrating lymphocyte (TIL) therapy has recently been approved by the US Food and Drug Administration and Health Canada for the management of patients with advanced melanoma refractory to first-line immune checkpoint inhibitors, with regulatory assessments underway in other jurisd…

DNA methylation (DNAm) plays a pivotal role in regulating gene expression and tissue function in the skin, which exhibits a high degree of responsiveness to environmental and lifestyle factors. These factors are believed to contribute to epigenetic drift, a hallmark of aging marked by increased meth…

Mitochondria are increasingly recognized as central regulators of vascular health, shaping endothelial cell function through roles that extend far beyond energy production. In addition to coordinating redox balance, calcium dynamics, and biosynthetic support, recent studies have revealed that mitoch…

Intercellular mitochondrial transfer has emerged as a fundamental mechanism of tissue adaptation and repair in the cardiovascular system, with major implications for cardiovascular, neurological, metabolic, and inflammatory diseases. Once thought to be static, mitochondria are now recognized as mobi…

Mitochondria are organelles that orchestrate numerous cell functions in addition to providing energy. During viral infection or in case of defects in mitochondrial replication, an intricate mechanism of self-destruction is engaged through the formation of mitochondrial pores. This leads to the relea…