bims-necame Biomed News
on Metabolism in small cell neuroendocrine cancers
Issue of 2025–11–16
25 papers selected by
Grigor Varuzhanyan, UCLA



  1. Trends Pharmacol Sci. 2025 Nov;pii: S0165-6147(25)00231-7. [Epub ahead of print]46(11): 1041-1043
      Small cell lung cancer (SCLC) progression relies on neuronal activity, yet the mechanisms remain unclear. Two recent studies by Savchuk et al. and Sakthivelu et al. reveal that SCLC co-opts vagal and sympathetic inputs and forms glutamatergic or GABAergic synapse-like connections with neurons, uncovering direct neuron-tumor crosstalk as a key driver of malignancy and a potential therapeutic vulnerability.
    Keywords:  nervous system; neuron–cancer crosstalk; receptors; small cell lung cancer; vagus nerve
    DOI:  https://doi.org/10.1016/j.tips.2025.10.007
  2. Commun Biol. 2025 Nov 13. 8(1): 1559
      Tumor cell plasticity in novel microenvironments is central to the integration and subsequent growth of metastatic cells. However, the functional consequences of tumor cell integration with central neurons remains understudied. Here, we address this question using small cell lung cancer (SCLC), which has an extraordinary propensity to metastasize to the brain in humans. Transcriptomic and electrophysiological analysis of SCLC cells in neuronal microenvironments reveal a heterogeneous population of synapse-forming SCLC cells with neurons. While a proportion of neuron-SCLC synapses are blocked by AMPA receptor antagonists, we also find a sensitivity of these synapses to GABAA receptor inhibition. The functional integration of SCLC with central neurons induced multiplicative synaptic upscaling between neurons and dysregulated neuronal excitability. Aberrant excitation in human neurons with SCLC was sustained by synaptic NMDA receptor activation and can be reduced by the FDA approved NMDA receptor blocker memantine. These findings reveal strategies to normalize tumor-induced exacerbation of aberrant neuronal activity.
    DOI:  https://doi.org/10.1038/s42003-025-09006-7
  3. Int J Mol Sci. 2025 Nov 06. pii: 10786. [Epub ahead of print]26(21):
      Small cell lung cancer (SCLC) is an aggressive neuroendocrine tumour with limited treatment options and a poor prognosis. Hypoxia, a hallmark of solid tumours, contributes to therapeutic resistance and tumour progression. Gastrin-releasing peptide receptor (GRPR) is known to be overexpressed in SCLC; however, its regulation under hypoxic conditions is not well described. In this study, we demonstrate that hypoxia significantly enhances GRPR expression in SCLC cell lines, COR-L24 and DMS79, as confirmed by Western blot, immunofluorescence, and flow cytometric analysis of binding with fluorescein isothiocyanate-labelled bombesin (BBN-FITC), a known GRPR ligand. To exploit this upregulation, we synthesised a previously discovered butylated neuropeptide antagonist (BU peptide) using a new method of solid-phase peptide synthesis (SPPS) by Boc chemistry and evaluated its therapeutic potential. BU peptide exhibited potent, dose-dependent cytotoxicity in both cell lines, with significantly greater efficacy under hypoxic conditions compared to normoxia. Mechanistic studies revealed that BU peptide inhibits GRP-GRPR-mediated activation of the PI3K/Akt and MAPK/ERK signalling pathways, known to be key regulators of tumour cell survival and proliferation. Moreover, BU peptide induced robust caspase 3/7-mediated apoptosis, especially under hypoxic conditions. These findings suggest that GRPR is a hypoxia-inducible target in SCLC and demonstrate that a synthetically optimised BU peptide antagonist exerts selective efficacy against hypoxic tumour cells, outperforming conventional chemotherapy agents. These findings provide new mechanistic insights into SCLC and suggest translational potential to inform the development of future treatment strategies for this and other hypoxia-driven malignancies.
    Keywords:  GRPR; hypoxia; neuropeptide antagonist; small cell lung cancer; targeted therapy
    DOI:  https://doi.org/10.3390/ijms262110786
  4. Front Oncol. 2025 ;15 1646608
      Small cell lung cancer (SCLC) is an aggressive neuroendocrine carcinoma with a poor prognosis and accounts for approximately 11% of all lung cancers. Owing to the complex and aggressive nature of the disease, clinical management of SCLC is challenging. Many SCLC regional guidelines, including those from East Asia, have been developed in light of potential regional variations in socioeconomic conditions and healthcare infrastructure. However, less is known about the potential implications of the inherent population/regional differences in clinical management and the emerging treatment landscape in SCLC. Here, we review variations in the real-world patient characteristics and in diagnosis and treatment guidelines in SCLC between East Asia and Europe/North America. We also consider similarities and differences in real-world treatment patterns, as well as clinical outcomes between regions, to explore the need to adapt clinical management in SCLC.
    Keywords:  Asian countries; East Asia; diagnosis; small cell lung cancer; survival outcomes; treatment guidelines; treatment patterns
    DOI:  https://doi.org/10.3389/fonc.2025.1646608
  5. Cell Oncol (Dordr). 2025 Nov 11.
      PURPOSE RETINOBLASTOMA L: (RB1) mutations frequently emerge as late subclonal events in advanced prostate cancer (PCa), driving inevitable recurrence and therapy resistance. Therapy-induced senescence (TIS) could promote metastasis at a late stage. However, the underlying mechanisms and therapeutic approaches for decetaxel-induced senescence (DIS) in RB1-deficient castration-resistant prostate cancer (CRPC) remain poorly understood.
    METHODS: We systematically evaluated the association between RB1 expression and tumor malignancy using TCGA-PRAD data and clinical prostate cancer samples. Multiple CRPC models were established, including RM-1 C57BL/6 and PC-3 BALB/c-nu mouse models, as well as human PC-3 and 22RV1 cells to uncover the double-edged nature of DIS. Subsequently, RNA sequencing of shRB1-DIS identified tumorigenic SASP factors. Furthermore, we investigated the molecular mechanisms of the combined treatment using techniques such as immunofluorescence, flow cytometry, chromatin immunoprecipitation (ChIP), dual luciferase reporter assay, and molecular docking.
    RESULTS: The clinical significance and negative correlation between RB1 expression and malignancy were verified in human PCa samples. Using murine and human CRPC models, we demonstrated that DIS response was retained in both RB1-knockdown and control groups. Strikingly, DIS promoted metastasis and accelerated the transition to neuroendocrine prostate cancer (NEPC) in RB1-deficient models. shRB1-DIS was marked by elevated senescence-associated β-galactosidase (SA-β-gal) activity and upregulation of p27Kip. RNA-seq analysis revealed a senescence-associated secretory phenotype (SASP) profile of shRB1-DIS, with upregulated IL-1α, CCL5, CCL20, MMP3, and IL-20. Mechanistically, we identified a novel FOXA1-IL20-IL20Rβ signaling axis which promoted macrophage polarization to M2-like phenotype. Notably, our data revealed that administration of ABT-263, eliminated shRB1 DIS-associated markers and SASPs, particularly, IL-20, both in vitro and in vivo experiments. Furthermore, molecular docking confirmed ABT-263 could directly bond to the IL-20 pocket with high affinity, and oeIL-20 advanced CRPC cells exhibited increased sensitivity to ABT-263 treatment. Therefore, the suppression of M2-like macrophages by ABT-263 was associated with reduced aggressiveness and decreased resistance to docetaxel in RB1-deficient CRPC.
    CONCLUSION: DIS accelerates the malignant progression of shRB1 CRPC, mediated by tumorigenic SASP, especially IL-20 enrichment. Notably, we identifies a novel FOXA1-IL-20-IL20Rβ axis that drives M2-like macrophage polarization and contributes to tumor aggressiveness and docetaxel resistance. Importantly, senolytic agent ABT-263 not only selectively eliminated shRB1-DIS cells but also restricted expression of tumorigenic SASPs, thereby restoring sensitivity to docetaxel. Wherein, IL-20 is inhibited through its interaction with ABT-263. These results provide a novel mechanistic rationale for using senolytic therapies to mitigate SASP-driven malignancy and improve treatment response in RB1-deficient CRPC.
    CLINICAL TRIAL NUMBER: Not applicable.
    DOI:  https://doi.org/10.1007/s13402-025-01126-w
  6. Proc Natl Acad Sci U S A. 2025 Nov 18. 122(46): e2513468122
      Epigenetic and transcriptional dysregulation plays a fundamental role in tumor lineage plasticity (LP). However, the underlying mechanisms, especially for the initial events of LP development, are still poorly understood. Here, we report that in progression of prostate cancer from adenocarcinoma to treatment-induced neuroendocrine prostate cancer (t-NEPC), anti-androgen receptor (AR) signaling inhibitors (ARSIs) reprogram the function of circadian regulator/nuclear receptor REV-ERBα by switching its target gene programs from kinase signaling and metabolic programs to programs of LP, which includes neurogenesis, stem cell, and epithelial-mesenchymal transition as well as over fifteen LP drivers including POU3F2/BRN2, ASCL1, FOXA2, ONECUT2, and MYCN. Unexpectedly, REV-ERBα facilitates the chromatin occupancy of BRN2, ASCL1, and FOXA1 in their activation of LP programs, thus functioning as a master regulator of ARSI-induced LP driver network. Mechanistically, REV-ERBα induces chromatin accessibility and H3K27ac modification at promoters of LP genes through its recruitment of BRD4 and p300. Overexpression of REV-ERBα alone is sufficient to induce LP and neuroendocrine phenotype and confers resistance to ARSI in adenocarcinoma cells. Loss of REV-ERBα potently inhibits NEPC cell growth and abolishes the expression of LP drivers and gene programs. Pharmacological inhibition of REV-ERBα exhibits high potency in blocking the growth of NEPC tumors including patient-derived xenografts. Our findings reveal that therapy-induced LP development entails a coordinated induction of a network of LP drivers and that REV-ERBα is an unexpected master regulator of the network and a promising therapeutic target for treatment of advanced prostate cancer such as NEPC.
    Keywords:  NEPC; REV-ERBα; drug resistance; lineage plasticity; therapeutic targeting
    DOI:  https://doi.org/10.1073/pnas.2513468122
  7. Front Oncol. 2025 ;15 1657441
      Small cell lung cancer (SCLC) is a highly aggressive malignancy characterized by early metastasis and poor prognosis due to the limited efficacy of current treatments. Although initially responsive to chemotherapy and radiotherapy, the majority of patients with SCLC develop resistance within a year, often succumbing to distant metastases. Historically, SCLC was considered a homogeneous disease, primarily driven by the deletion or inactivation of key tumor suppressor genes TP53 and RB1. However, recent advancements in genomics and single-cell sequencing have identified distinct molecular subtypes of SCLC, derived from studies on cell lines, animal models, and tumor tissues. The tumor's complexity, marked by the coexistence of multiple dynamic subtypes, contributes to its pronounced heterogeneity. Notably, different subpopulations exhibit a complex spatial relationship characterized by both mutual exclusion and coexistence. Temporally, SCLC exhibits the ability to undergo subtype transformations through various molecular mechanisms, underscoring the tumor's plasticity and offering novel perspectives for personalized treatment approaches. This review synthesizes recent discoveries regarding SCLC subtype classification, intratumor heterogeneity, plasticity-related signaling pathways, immune landscape, and emerging therapeutic strategies.
    Keywords:  ASCL1; neuroD1; neuroendocrine; plasticity; small cell lung cancer; tumor heterogeneity; tumor microenvironment
    DOI:  https://doi.org/10.3389/fonc.2025.1657441
  8. Cancer Treat Rev. 2025 Nov 04. pii: S0305-7372(25)00166-5. [Epub ahead of print]141 103044
      EGFR-mutant lung adenocarcinoma (LUAD) that transforms into small-cell lung cancer (SCLC) following targeted therapy represents a clinically significant mechanism of acquired resistance, occurring in approximately 3-14 % of cases. This transformed variant, referred to as SCLC after transformation (SCLC-AT), follows an aggressive clinical course and carries a poor prognosis. SCLC-AT retains the original EGFR mutation but significantly down-regulates EGFR protein expression, eliminating its dependence on EGFR signaling while simultaneously acquiring a neuroendocrine phenotype. In nearly all cases, bi-allelic inactivation of both TP53 and RB1 is observed. In this overview, we provide a detailed examination of the molecular mechanisms underlying the transition from EGFR-mutant LUAD to SCLC. By integrating two primary biological concepts, clonal evolution and lineage reprogramming, we examine recent advances concerning the original cell or cells involved, as well as the genetic and epigenetic reprogramming and signaling pathway changes. While the transformation to SCLC is currently considered genetically and epigenetically irreversible, preclinical interventions targeting EZH2, AURKA, and DLL3 have shown potential in reversing the neuroendocrine phenotype or improving tumor immunogenicity. Future research should utilize single-cell and spatial multi-omics technologies to develop predictive models that can facilitate the early detection of impending transformation and guide precision therapies, ultimately enhancing patient outcomes.
    Keywords:  EGFR; Lung adenocarcinoma; Small-cell lung cancer; Therapeutic resistance; Transformation
    DOI:  https://doi.org/10.1016/j.ctrv.2025.103044
  9. Front Endocrinol (Lausanne). 2025 ;16 1680209
       Background: The prevalence and clinical relevance of bone metastases (BM) in advanced small intestinal neuroendocrine tumors (siNETs) is not well-documented.
    Methods: We analyzed data from 458 patients (54% male, median age 58 years) with histologically confirmed siNETs treated at the ENETS Center of Excellence Essen from 2003 to 2023. BM occurrence and their impact on skeletal-related events (SREs) and overall survival (OS) were assessed using standardized DOTATOC-PET/CT within a consistent "one-stop shop" multidisciplinary care model.
    Results: At diagnosis, 305/458 patients (66.6%) had stage IV disease; BM were detected in 105/305 (34.4%). Functioning tumors were more frequent in BM patients (73%) than in the total cohort (40%). In 48.6% of patients, BM were initially visible on SSTR imaging only, becoming morphologically detectable after a median of 16 months. Most BM were osteoblastic (58%). During a median follow-up of 36 months, SREs occurred in 12.4% of BM patients, predominantly in those with osteolytic disease. SREs occurred in 27% of patients without antiresorptive therapy, but in none with treatment (p < 0.0001). Median OS was significantly shorter in patients with BM (127 vs. 170 months, p = 0.023), independent of age, sex or tumor grade.
    Conclusion: BM are frequent in siNET, particularly in functioning tumors, and are associated with reduced survival. BM may initially be detectable only by functional imaging but becomes morphologically visible within less than 1.5 years. Antiresorptive therapy may reduce SREs. Whether adapting NET treatment algorithm for BM improves OS needs to be tested in clinical trials.
    Keywords:  DOTATOC-PET/CT; bone metastases; functioning NET; ileum NET; neuroendocrine tumors; small intestine
    DOI:  https://doi.org/10.3389/fendo.2025.1680209
  10. Clin Case Rep. 2025 Nov;13(11): e71464
      Rectal neuroendocrine neoplasms are rare, aggressive tumors comprising a small fraction of rectal malignancies but with increasing incidence due to improved endoscopic detection. Small cell neuroendocrine carcinoma, a high-grade variant, poses significant diagnostic and therapeutic challenges due to its rapid progression and poor prognosis. We report a case of a 32-year-old male presenting with per-rectal bleeding and pain for 6 months. Imaging revealed an anorectal mass with locoregional lymphadenopathy. Histopathology confirmed poorly differentiated small cell neuroendocrine carcinoma (Grade 3) via immunohistochemistry, with a high Ki-67 index and neuroendocrine marker expression. The patient received neoadjuvant radiotherapy (59.4 Gray in 30 fractions over 6 weeks) with concurrent chemotherapy etoposide and cisplatin, followed by abdominoperineal resection. Postoperative histology showed a pathological complete response. This case underscores the importance of considering rectal neuroendocrine neoplasms in patients with persistent rectal symptoms and highlights the role of immunohistochemistry in diagnosis. Despite limited literature, our case supports the potential efficacy of neoadjuvant radiochemotherapy in tumor downstaging and improving surgical outcomes in poorly differentiated rectal neuroendocrine carcinoma. Early diagnosis, protocol-based neoadjuvant radiochemotherapy, and appropriate surgical intervention can lead to favorable outcomes in high-grade rectal neuroendocrine neoplasms. This case reinforces the need for awareness, guideline-based management, and long-term surveillance to optimize prognosis in these rare but aggressive tumors.
    Keywords:  abdominoperineal resection; neuroendocrine; radiochemotherapy; rectal bleed; small cell carcinoma
    DOI:  https://doi.org/10.1002/ccr3.71464
  11. J Thorac Dis. 2025 Oct 31. 17(10): 7826-7837
       Background: Clinical research and decision-making for small cell lung cancer (SCLC) are still based on the dichotomous classification of limited-stage disease or extensive-stage disease. This study aimed to evaluate the prognosis of the 9th edition of the tumor-node-metastasis (TNM) classification for SCLC in a real-world setting.
    Methods: This retrospective study included SCLC patients diagnosed between January 2014 and December 2021 in our center. Patients who had undergone complete staging evaluation were re-staged according to the 8th and 9th editions of the TNM classification for lung cancer. Kaplan-Meier analysis was conducted to determine overall survival (OS). Univariate and multivariate analyses were performed using the Cox proportional hazards model to assess the prognostic impact of the relevant factors.
    Results: A total of 1,329 patients were included in the study. All patients underwent clinical staging, while pathological staging was available on 51 patients. After a median follow-up of 39.67 months [95% confidence interval (CI): 36.28-43.06], the median OS was 15.40 months (95% CI: 14.45-16.35). According to the 8th and 9th TNM classification, the median OS, 1-, 2-, and 3-year OS rates showed a progressive decline with advancing stage. The median OS of the N2a and N2b subclasses that were newly added to the 9th edition was 20.00 months (95% CI: 16.14-23.86) and 14.53 months (95% CI: 12.88-16.18), respectively. The 3-year survival rates were 22.9% and 14.6%, respectively. Patients with N2b exhibited poorer outcomes (P=0.002). However, compared to M1c2, the median OS was numerically longer in patients with M1c1 (11.67 vs. 10.63 months), but it did not achieve a statistically significant difference (P=0.13). Multivariate analysis revealed that both the 8th and 9th editions of the TNM were independent prognostic factors.
    Conclusions: This retrospective analysis confirmed a significant difference in prognosis between the N2a and N2b subgroups in the 9th edition. No statistically significant difference was observed between the M1c1 and M1c2 subgroups. The present study suggests that the 9th edition of TNM requires further validation using external datasets, and factors such as the number of metastatic lesions in a single organ may also be included in future TNM classification systems.
    Keywords:  9th edition of the tumor-node-metastasis (9th TNM edition); Small cell lung cancer (SCLC); applicability; prognosis
    DOI:  https://doi.org/10.21037/jtd-2025-1031
  12. Sci Rep. 2025 Nov 14. 15(1): 39993
      To validate and compare conventional metabolic tumor burden measurements with comprehensive metabolic tumor distribution patterns using [18F] fluorodeoxyglucose positron emission tomography/computed tomography (FDG PET/CT) to predict small-cell lung cancer (SCLC) prognosis. This retrospective study included 520 patients with SCLC (mean age ± standard deviation, 67 ± 5.6 years; 84.8% men) who underwent PET/CT for staging. Of these, 364 scans were used for training (n = 291) and internal (n = 73) tests, while 156 other scans were used for external testing. Clinical data (age, sex, and stage) were reviewed. Volumes of interest were manually drawn using a threshold standard uptake value of 2.5 for total lesion glycolysis (TLG) for all tumor lesions on PET. TLG with distribution (TLGd) and organ-based tumor distribution (metastasis in organs, METAORG) was analyzed from CT-based automatic organ segmentation and overlaid on PET. Four survival prediction models (event and duration) were developed using a Random Forest classifier: (1) clinical factors, (2) tumor TLG, (3) TLGd and METAORG, and (4) combined models. The top 11 features were selected for survival duration prediction included clinical factors (age and stage), TLG, five TLGd radiomics features, and three METAORG features (axial and peripheral skeletal distribution patterns and the liver distribution pattern). In the internal test, C-indices for overall survival were 0.611, 0.592, 0.721, and 0.753 for tumor TLG, clinical, METAORG, and combined model, respectively. External test C-indices were 0.637, 0.326, 0.706, and 0.740, respectively. The combined model, which incorporated tumor distribution information such as TLGd and METAORG, demonstrated the highest predictive power for both test sets. The combined model outperformed the other models in predicting survival. Application of tumor distribution (TLGd and METAORG) to whole-body tumor distribution pattern analysis shows promise for improving prognosis evaluation, with advantages of quantifiable metastasis stratification.
    Keywords:  Quantitative imaging; Small-cell lung cancer; Tumor burden; Tumor distribution; [18F] FDG PET/CT
    DOI:  https://doi.org/10.1038/s41598-025-23649-w
  13. Transl Lung Cancer Res. 2025 Oct 31. 14(10): 4682-4687
       Background: Spontaneous regression (SR) of small cell lung cancer (SCLC) is an extremely rare phenomenon, and its underlying mechanisms remain unclear. Previous reports have suggested that immune-related factors, including paraneoplastic neurological syndrome (PNS) and anti-Hu antibodies, may be involved. In this report, we present a case of SCLC that demonstrated SR after initial progression, and explore the potential immunological mechanisms, particularly focusing on changes in the tumor microenvironment.
    Case Description: A 78-year-old Japanese male with a significant smoking history presented with two pulmonary nodules. Surgical resection revealed non-small cell lung cancer (NSCLC) and SCLC (stage IA2). Seven months later, SCLC recurrence was confirmed via transbronchial needle aspiration (TBNA) of the subcarinal lymph nodes. First-line chemotherapy with carboplatin and etoposide resulted in a good response; however, disease progression was observed later. Second-line treatment with carboplatin and nanoparticle albumin-bound (nab)-paclitaxel was initiated, but therapy was then discontinued because of declining performance status. Unexpectedly, without further treatment, the tumor and serum gastrin-releasing peptide precursor (pro-GRP) levels began to decrease over 6 months, indicating SR. Immunohistochemical analysis revealed an increase in cluster of differentiation (CD) 8-positive T cells and a decrease in regulatory T cells in the recurrent tumor, thus suggesting immune modulation.
    Conclusions: This case of SCLC showed SR following chemotherapy, which was likely influenced by immune-related mechanisms. Changes in the tumor microenvironment, particularly an increase in cytotoxic T cells and a reduction in regulatory T cells, may contribute to SR. Nab-paclitaxel may contribute to the modulation of the tumor immune microenvironment. Therefore, understanding these mechanisms could provide insights into tumor immunity and potential therapeutic strategies.
    Keywords:  CD8-positive T cells; Small cell lung cancer (SCLC); case report; nab-paclitaxel; spontaneous regression (SR)
    DOI:  https://doi.org/10.21037/tlcr-2025-578
  14. J Neuroendocrinol. 2025 Nov 15. e70112
      Somatostatin analogs (SSAs) are an established first-line therapy in intestinal and pancreatic neuroendocrine tumors (NETs). Based on Phase III studies, their use is recommended in NET with a Ki-67 index of up to 10%. The effect of first-line SSA therapy on differentiated NET with a Ki-67 index ≥10% is poorly understood. This study aimed to investigate the outcomes of SSA therapy in differentiated NETs with a Ki-67 index of ≥10%. A retrospective analysis of a prospectively created dataset of consecutive patients with NETs was performed. Patients with first-line SSA monotherapy in advanced NET with a Ki-67 index ≥10% were included. The study endpoints were progression-free survival (PFS), overall survival (OS), and clinical benefit rate, defined as partial remission (PR) or stable disease (SD). Of 362 consecutive patients with a Ki-67 index ≥10%, 67 received first-line SSA therapy. The Ki-67 index was 10-20% (G2) in 57 (85%) patients and >20% (G3) in 10 (15%). SD as the best response was reached in 40 (59.7%) patients and PR in 3 (4.5%), irrespective of the NET origin, time from the diagnosis, or somatostatin receptor-based tracer uptake. The median PFS was 18 (95% confidence interval [CI], 5.7-30.3) months, and the median OS was 60 (95% CI, 38.2-81.8) months after the initiation of SSA therapy. Median PFS was significantly longer in patients with a Ki-67 index of 10-20% (19 months; 95% CI, 6.2-31.8) compared to those with G3 NETs (6 months; 95% CI, 2.9-9.1; p = .015, log-rank test), and in patients with a liver tumor burden of ≤10% (24 months; 95% CI, 12.7-35.3) versus >10% (4 months; 95% CI, 2.3-5.7; p = .007). First-line SSA therapy can provide meaningful disease control in patients with G2 NETs and low tumor burden, despite a Ki-67 index ≥10%. It may be a reasonable alternative to more intensive therapies in selected patients.
    Keywords:  Ki‐67 index ≥10%; neuroendocrine tumor G2; neuroendocrine tumor G3; somatostatin analogues in NET
    DOI:  https://doi.org/10.1111/jne.70112
  15. Front Oncol. 2025 ;15 1645589
       Background: The heterogeneity of the lung cancer brain metastasis (LCBM) microenvironment limits therapeutic efficacy, while invasive pathological biopsies fail to dynamically assess brain metastasis (BM) comprehensively. Non-invasive imaging techniques thus hold clinical value for visualizing the LCBM microenvironment. This study aimed to achieve non-invasive quantitative analysis of vascular function and cellular structures in LCBM using multiparametric Dynamic Contrast-Enhanced Magnetic Resonance Imaging (DCE-MRI) and Diffusion-Weighted Imaging (DWI).
    Methods: A prospective cohort of 114 LCBM patients (63 lung adenocarcinoma [LUAD]-BM, 28 lung squamous cell carcinoma [LUSC]-BM, 23 small cell lung cancer [SCLC]-BM) underwent DCE-MRI and DWI on a 3.0T MRI scanner. Parameters including volume transfer constant (Ktrans), rate constant (Kep), extravascular extracellular volume (Ve), and plasma volume fraction (Vp) were derived using the Extended Tofts model. Group differences were analyzed via Mann-Whitney U test, diagnostic efficacy via ROC curves, and parameter interactions via multivariate logistic and linear regression.
    Results: ADC distinguished SCLC-BM from NSCLC-BM with AUC=0.891 (specificity=95.05% at 752.4×10-6mm²/s), while Ktrans differentiated LUAD-BM from LUSC-BM with AUC=0.998 (sensitivity=98.48%, specificity=97.79% at 157 min-1/1000). Microenvironmental profiles: LUAD-BM showed high Vp (51.50/1000) and Ktrans (424.8 min-1/1000); LUSC-BM had low Ktrans (61.15 min-1/1000) and medium ADC (1163×10-6mm²/s); SCLC-BM exhibited high cellular density (ADC=661×10-6mm²/s) and abnormal contrast kinetics (high Kep, low Vp). Vp and ADC were identified as independent predictors for LUAD-BM and SCLC-BM, respectively. Parameter interactions varied by subtype: ADC in LUAD-BM correlated with Kep and Delay Time; in LUSC-BM, with Ktrans and Ve; and in SCLC-BM, showed weaker vascular associations. Ktrans regulation involved distinct parameter contributions across subtypes.
    Conclusion: A DCE-MRI-DWI "Vascular-Cellular Microenvironment Visualization Model" was established, revealing distinct profiles: high microvascular density/permeability in LUAD-BM, low permeability/medium cellularity in LUSC-BM, and high cellularity/abnormal contrast kinetics in SCLC-BM. This validates multimodal imaging for characterizing LCBM heterogeneity and provides insights into tumor angiogenesis, cellular density, and BBB regulation, supporting microenvironment-targeted therapy.
    Keywords:  brain metastasis (BM); diffusion-weighted imaging (DWI); dynamic-contrast-enhanced magnetic resonance imaging; lung cancer; microenvironment
    DOI:  https://doi.org/10.3389/fonc.2025.1645589
  16. Ther Adv Med Oncol. 2025 ;17 17588359251389742
       Background: Systemic therapy is a standard treatment option for pancreatic neuroendocrine neoplasms (Pan-NENs) with unresectable or metastatic disease. Streptozocin (STZ)-based chemotherapy is considered a standard treatment option for tumors with a high Ki-67 index or for cases refractory to molecular targeted agents. More recently, the combination of capecitabine and temozolomide (CAPTEM) therapy has emerged as a new treatment option.
    Objectives: This study aimed to compare the efficacy, safety, and clinical outcomes between the STZ-based regimen and CAPTEM therapy in patients with unresectable or metastatic Pan-NENs.
    Design: This was a single-center retrospective study of histologically confirmed Pan-neuroendocrine tumor (NET) patients treated with either STZ-based regimens or CAPTEM between November 2015 and June 2024.
    Methods: We compared efficacy, safety, and clinical outcomes between the two regimens. Tumor responses were assessed using Response Evaluation Criteria in Solid Tumors version 1.1, and adverse events were graded according to Common Terminology Criteria for Adverse Events version 5.0. The study was conducted in compliance with the STROBE guidelines.
    Results: Of the 371 patients diagnosed with Pan-NENs, 47 received STZ-based regimen and 21 received CAPTEM therapy. In the NET-G1/G2 patients, the STZ group showed a significantly higher tumor shrinkage rate compared to CAPTEM therapy. Although no significant differences were observed in progression-free survival (PFS) or overall survival between the two groups, subgroup analysis showed that the median PFS in the STZ group was significantly longer than that in the CAPTEM group in NET G1/G2 patients. Renal dysfunction was the main adverse event in the STZ regimen group, while gastrointestinal symptoms were common in the CAPTEM therapy group; however, both were manageable.
    Conclusion: Both STZ-based regimen and CAPTEM therapy are safe and effective treatment options for advanced Pan-NENs. STZ-based regimen was more beneficial in NET-G1/G2 patients, suggesting Ki-67 index and tumor grade may serve as indicators for treatment selection. Further prospective studies are warranted to validate these findings.
    Keywords:  capecitabine and temozolomide; chemotherapy; ki-67 index; pancreatic neuroendocrine neoplasm; streptozocin
    DOI:  https://doi.org/10.1177/17588359251389742
  17. World J Nucl Med. 2025 Sep;24(3): 244-252
      Inspired by international frameworks, New Zealand established a nationally coordinated peptide receptor radionuclide therapy service. This article reflects on the key steps involved in building the service, including the formation of a national neuroendocrine tumor (NET) multidisciplinary meeting, the role of patient advocacy, and the integration of local research. The successful creation of the service, despite significant challenges, demonstrates the value of collaboration between clinicians, government, universities, and patient groups in achieving equitable, high-quality care.
    Keywords:  NET; New Zealand; PRRT; health equity; implementation; multidisciplinary
    DOI:  https://doi.org/10.1055/s-0045-1807726
  18. Gastroenterology. 2025 Nov 14. pii: S0016-5085(25)05740-3. [Epub ahead of print]
      Neuroendocrine tumors (NETs) represent a heterogeneous group of neoplasms with diverse biological and clinical behavior, and gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are the most common subtype. This review provides an overview of GEP-NETs, with a focus on incidence trends, pathologic classification, diagnostic strategies, therapeutic advances, and the role of endoscopy in diagnosis and management of GEP-NETs. Incidence rates of GEP-NETs have significantly increased over recent decades, largely due to improved diagnostic modalities and increased use of endoscopy, although environmental factors may be at play as well. Pathologic grading and classification, based on the 2022 World Health Organization criteria, remain essential in defining prognosis and therapeutic options, and advanced imaging modalities like somatostatin receptor positron emission tomography scans enable precise localization and staging. Therapeutic approaches vary by tumor grade, stage, and localization, from an increasing role for endoscopic management of indolent tumors to surgical resection for certain higher-risk subtypes and for resectable metastatic disease. Novel treatments, including somatostatin analogs, radioligand therapy, mammalian target of rapamycin inhibitors, and antiangiogenic agents, have shown significant efficacy in advanced and metastatic disease. Future research is needed to identify molecular markers to refine diagnostic accuracy and personalize treatment strategies, thereby improving long-term outcomes for GEP-NET patients. Additionally, research is needed to better define GEP-NET subtypes that are appropriate for endoscopic therapy as well as to understand long-term outcomes after endoscopic resection. Given the important role that endoscopy has in the diagnosis and management of GEP-NETs, increased recognition of and knowledge about these tumors is critical for the gastroenterology community.
    Keywords:  Endoscopic Management; Gastroenteropancreatic Neuroendocrine Tumors; Neuroendocrine Tumors; Recommendations
    DOI:  https://doi.org/10.1053/j.gastro.2025.07.004
  19. Virchows Arch. 2025 Nov 14.
      Neuroendocrine neoplasms (NENs) are a heterogeneous group of tumors. The rarity of the disease, together with the lack of mutations in the classical tumor suppressor genes and the paucity of models, has impaired our understanding of the mechanisms of progression and the cell of origin of these tumors. Due to their higher frequency, this review focuses on Gastro-Entero-Pancreatic (GEP) and Lung NENs. While recent molecular profiling has shed light on the possible cell of origin of GEP- and lung NENs, many questions remain unanswered and further studies using proper in vitro and in vivo models are needed, combined with the latest technologies such as single-cell and spatial sequencing and deep-learning for digital pathology. Genomic and epigenomic evidence suggests that pancreatic NENs originate from adult pancreatic cells rather than common progenitor cells; however, ultimate proof in vitro or in vivo is still lacking. Similarly, emerging molecular evidence suggests that lung NENs may have very diverse origins, encompassing most lung cell types, but much work is still needed to pinpoint their cell of origin. Further, tumors with mixed endocrine and non-endocrine composition suggest the possibility of trans-differentiation and acquisition of neuroendocrine features in different cell types. This review aims to summarize emerging insights on this topic, highlight future directions for identifying the cell of origin of NENs in these organs and explore how this knowledge may ultimately translate into clinical advances.
    Keywords:  Carcinoma; Cell of origin; Gastro-entero-pancreatic endocrine neoplasm; Lung neuroendocrine cancer; Neuroendocrine neoplasms
    DOI:  https://doi.org/10.1007/s00428-025-04311-2
  20. Endocr Pathol. 2025 Nov 10. 36(1): 44
      About 40% of non-functioning (NF) Pancreatic Neuroendocrine Tumors (PanNETs) harbour mutations in MEN1, often co-occurring with DAXX/ATRX. While the ADM group (MEN1 and DAXX/ATRX co-mutated) exhibits homogeneous genetic and epigenetic features and a consistent risk of relapse, it shows considerable variability in treatment response, suggesting an underlying molecular diversity. In this study we aimed to elucidate the molecular mechanisms underlying this heterogeneity of ADM PanNETs by integrating transcriptomic (n = 36) and DNA methylation (n = 93) data. First, DNA methylation discriminates ADM-PanNET from PanNET mutated only in MEN1 (α-like), revealing enhancer, peri-centromeric, and telomeric methylation changes associated with alternative lengthening of telomeres and increased chromosomal instability. Transcriptomic analysis further revealed three distinct ADM subtypes: ADM hypoxic, ADM NST (No Special Type), and ADM immunosuppressive. The ADM hypoxic subtype is characterized by strong hypoxia signature, likely regulated epigenetically. The ADM NST subtype appears to be primarily driven by epigenetic changes that promote proliferation. Notably, the ADM immunosuppressive subtype, although significantly smaller (< 2.5 cm, p = 0.023), exhibits strong immune and metastasis-like signatures, suggesting a uniquely aggressive biology despite its reduced size. By defining these three novel ADM subtypes, our study provides a refined framework for understanding PanNET heterogeneity. This classification underscores potential diagnostic markers and highlights distinct biological vulnerabilities that may inform the development of subtype-tailored therapeutic strategies.
    Keywords:  Epigenome; Neuroendocrine; Pancreas; Transcriptome; Tumor
    DOI:  https://doi.org/10.1007/s12022-025-09889-6
  21. AME Case Rep. 2025 ;9 154
       Background: Small cell neuroendocrine carcinomas (SCNECs) of the gastrointestinal (GI) tract are exceedingly rare and aggressive malignancies. Unlike pulmonary SCNECs, which are well-characterized, extrapulmonary GI-SCNECs lack established diagnostic pathways and treatment algorithms due to their rarity and histopathological overlap with other poorly differentiated tumors. Literature on their clinical course remains limited to isolated reports, with scarce data on site-specific presentations. These two cases contribute to the existing literature by illustrating distinct primary GI sites (gastric and rectal), underscoring the challenges in early diagnosis and the aggressive clinical progression despite intervention.
    Case Description: The first patient, an 80-year-old man, presented with progressive dysphagia and dyspnea. Imaging revealed mediastinal lymphadenopathy and gastric wall thickening. Endoscopic biopsies confirmed gastric SCNEC with widespread metastases. Despite initiation of palliative care, his condition deteriorated rapidly, and he died one month after diagnosis. The second patient, a 69-year-old man, presented with significant weight loss and lower abdominal pain. Colonoscopy identified a circumferential rectal mass, and biopsies confirmed SCNEC. Staging demonstrated hepatic and nodal metastases. He received platinum-based chemotherapy and pelvic radiotherapy, achieving only transient stabilization. Following treatment cessation, his disease progressed, and despite resuming chemotherapy, he died 11 months after diagnosis.
    Conclusions: These cases emphasize the necessity for heightened clinical suspicion of SCNEC in rapidly progressive GI malignancies, even in atypical presentations. Early recognition and multidisciplinary management are crucial, although current therapeutic options offer limited durability. By delineating site-specific clinical courses, this report contributes to the limited body of evidence on GI-SCNECs and underscores the urgent need for standardized diagnostic criteria and treatment protocols to improve outcomes.
    Keywords:  Small cell carcinoma; case report; gastric ulcer; gastrointestinal neuroendocrine carcinoma (GI-NEC); rectal mass
    DOI:  https://doi.org/10.21037/acr-2025-71
  22. Transl Cancer Res. 2025 Oct 31. 14(10): 7457
    Editorial Office
      [This corrects the article DOI: 10.21037/tcr-2024-2482.].
    DOI:  https://doi.org/10.21037/tcr-2025b-18
  23. Endocr Relat Cancer. 2025 Nov 12. pii: ERC-25-0272. [Epub ahead of print]
      The CUT/Hox transcription factor ONECUT2 (OC2) promotes lineage plasticity and is a confirmed therapeutic target in prostate cancer and several other malignancies where cell phenotype plays a substantial role in treatment resistance. OC2 governs a broad growth and lineage identity process in prostate cancer that promotes neuroendocrine (NE) differentiation, androgen receptor (AR) suppression, and the emergence of a wide range of treatment-resistant pathways. The mode of action of OC2 includes incorporation of the protein into transcription complexes at gene promoters as an activator and repressor, alteration of chromatin accessibility and epigenetic marks, and extensive alteration of large-scale chromatin modifications, such as super-enhancers and chromatin loops. Notably, OC2 may be unique among NE drivers in that it can promote AR-indifference in adenocarcinoma as a direct upstream activator of the glucocorticoid receptor, thus assuming indirect control of a portion of the AR cistrome. OC2 expression and activity increase substantially following hormone therapy in association with aggressive disease in prostate and breast cancer. Experiments in model systems have shown that OC2 has a survival function in both human castration-sensitive and castration-resistant prostate cancer cells. OC2 can be targeted directly with a family of novel small molecule inhibitors that show therapeutic efficacy in vivo in prostate, breast and gastric cancer models, including regression of established distant metastases in mice. These findings suggest that inhibition of OC2 clinically may confer substantial therapeutic benefit in some aggressive malignancies, including in localized, hormone-sensitive disease.
    DOI:  https://doi.org/10.1530/ERC-25-0272
  24. J Endocrinol Invest. 2025 Nov 12.
    NIKE Group
      Multiple endocrine neoplasia type 1 (MEN1) is an autosomal dominant disorder caused by MEN1 gene mutations, typically involving primary hyperparathyroidism (PHPT), pancreatic neuroendocrine tumors (PanNETs), and/or pituitary neuroendocrine tumors (PitNETs). However, 10-30% of patients with MEN1-like features lack identifiable MEN1 mutations and are classified as phenocopies. This retrospective multicenter study, conducted across 10 Italian referral centers, aimed to characterize the main clinical features of phenocopies. Among 240 patients evaluated for suspected MEN1 over five years, 175 (mean age 43.2 ± 19.7; 101 females) had genetically confirmed MEN1, while 65 (27%; mean age 59.9 ± 11.6; 44 females) were identified as phenocopies. Of these, 46 (70.7%) were also negative for CDKN1B mutations, confirming the rarity of MEN4. Phenocopies were diagnosed one to two decades later than MEN1 patients (p < 0.0001). PHPT was the most frequent manifestation in both groups (80% of phenocopies vs. 81% of MEN1), but tumor associations differed significantly between groups (p < 0.001): 41% of MEN1 patients showed the classic triad, compared to only 1% of phenocopies; PHPT with NETs was more common in MEN1 (32%), whereas PHPT with PitNETs occurred more often in phenocopies (54%), reflecting patterns of sporadic tumors. Notably, 11% of phenocopies had a first-degree relative with MEN1-related diseases, and 51% had a personal or family history of cancer. In conclusion, MEN1 phenocopies are relatively common and represent a clinical challenge. Given their distinct features and familial backgrounds, an extended genetic panel should be offered to these patients together with periodical screening of MEN1-related disease.
    Keywords:  Genetic analysis; Multiple endocrine neoplasia type 1 (MEN1); Neuroendocrine tumors; Phenocopy; Pituitary adenomas; Primary hyperparathyroidism
    DOI:  https://doi.org/10.1007/s40618-025-02743-w