bims-ovdlit Biomed News
on Ovarian cancer: early diagnosis, liquid biopsy and therapy
Issue of 2026–03–29
ten papers selected by
Lara Paracchini, Humanitas Research



  1. Acad Oncol. 2025 ;2(1):
      Among human malignancies, ovarian epithelial neoplasms are unique because they are unlikely to develop from their cognate organs, the ovaries (specifically, the surface mesothelium). The updated paradigm about the origin of high-grade serous carcinoma (HGSC) suggests that many HGSCs derive from the fallopian tubes following a sequential tumor progression, from pathologically defined p53 signature, serous tubal intraepithelial lesion, and serous tubal intraepithelial carcinoma (STIC) to HGSC that later spreads to ovarian tissues and disseminates. Despite the fact that the biological and clinical significance of each of those precursor lesions is yet to be elucidated, molecular and morphological correlative studies demonstrate unique features associated with various precancerous lesions. Chromosomal instability, aneuploidy patterns, and the activation of specific cancer signaling pathways attribute tumor progression to HGSC. The knowledge gained thus far is transforming various aspects of ovarian cancer research and gynecological practice. Opportunistic salpingectomy prevents HGSC in average-risk women, and molecular analyses in routine liquid-based cervical Pap tests hold promise to detect STIC- and HGSC-related tissue biomarkers. This review article will summarize those key findings in the earliest development of HGSC precursors and discuss the future challenges and promises of translating this paradigm shift to leverage standardization in diagnostic, early detection, and prevention of this devastating ovarian cancer.
    Keywords:  BRCA mutation; CCNE1 and MYC amplification; aneuploidy; ovarian high-grade serous carcinoma; p53 mutation; serous tubal intraepithelial carcinoma
    DOI:  https://doi.org/10.20935/acadonco7620
  2. Fam Cancer. 2026 Mar 24. pii: 33. [Epub ahead of print]25(2):
      
    Keywords:  Early detection of cancer; Endometrial cancer; Lynch syndrome; Ovarian cancer; Prevention of cancer
    DOI:  https://doi.org/10.1007/s10689-026-00548-1
  3. medRxiv. 2026 Mar 06. pii: 2026.03.05.26347746. [Epub ahead of print]
      Accurate classification of BRCA1 and BRCA2 variants is essential for cancer risk assessment and therapy selection, yet over one-third remain variants of uncertain significance (VUS). Here, using 120,660 real-world cancer genomic profiles with BRCA1 or BRCA2 variants from a >800,000-sample cohort, we develop machine learning models that predict pathogenicity using clinical and tumor-derived features, including a pan-cancer homologous recombination deficiency signature, co-mutated genes, zygosity, and cancer type. Trained on classified variants from ClinVar, our models achieved near-perfect performance, with validation ROC-AUC of 1.000 for BRCA1 and 0.989 for BRCA2 variants with ≥5 observations, translating to strong benign or pathogenic evidence for VCEP classification. Applying these models to 1,073 BRCA1 and 1,639 BRCA2 VUS, we strengthened or enabled classification of 39.48% BRCA1 and 50.52% BRCA2 assessable variants. This approach transforms underutilized tumor profiling data into evidence that can be directly integrated into variant classification, providing a scalable framework for other tumor profiling datasets and cancer genes associated with defined tumor genomic features.
    DOI:  https://doi.org/10.64898/2026.03.05.26347746
  4. Cancer Biol Med. 2026 Mar 24. pii: j.issn.2095-3941.2025.0619. [Epub ahead of print]
      Ovarian cancer was the eighth most frequently diagnosed cancer among women in 2022. The global age-standardized incidence rate of ovarian cancer decreased from 7.22/100,000 to 6.71/100,000 from 1990 to 2021. However, incidence trends varied across countries. Declining ovarian cancer incidence rates were reported in high-income countries, such as the United States, Austria, the Netherlands, and Norway, while there were increasing incidence rates in Africa and parts of Asia, including Japan and India. The global age-standardized mortality rate of ovarian cancer decreased from 4.73/100,000 to 4.06/100,000 between 1999 and 2021 with varying trends among countries. Moreover, the age-standardized 5-year net ovarian cancer survival rate in most countries remained < 50%. Several specific factors related to ovarian cancer risk have been identified, including reproductive factors, use of oral contraceptives, anti-inflammatory diets, endometriosis, pelvic inflammatory disease, obesity, diabetes, and occupational asbestos exposure. No screening or prevention strategy has been proven effective in downstaging or reducing mortality from ovarian cancer in an average-risk population without a family cancer history or pathogenic variants. Indeed, risk-reducing salpingo-oophorectomy remains the gold standard for lowering the risk of ovarian cancer in high-risk individuals with hereditary mutations. This review provides a comprehensive overview of the epidemiology, risk factors, screening, and prevention of ovarian cancer, aiming to offer a global perspective on public health strategies for addressing the disease.
    Keywords:  Ovarian cancer; epidemiology; prevention; risk factors; screening; trend
    DOI:  https://doi.org/10.20892/j.issn.2095-3941.2025.0619
  5. Clin Transl Med. 2026 Mar;16(3): e70594
      Early detection of gastrointestinal (GI) cancers remains a critical unmet clinical need, as most patients are diagnosed at advanced stages when prognosis is poor. Liquid biopsy has emerged as a transformative approach for minimally invasive cancer detection by analysing tumour-derived analytes in blood and other body fluids. Recent advances in circulating tumour DNA (ctDNA) sequencing, cell-free DNA methylation profiling, fragmentomics, extracellular vesicle and exosome characterisation, circulating tumour cell isolation and tumour-educated platelets have markedly improved sensitivity and specificity for detecting incipient malignancies. Despite these advances, sensitivity in stage I disease remains limited due to low tumour burden and minimal analyte scaling, resulting in false-negative results for small or indolent lesions. In addition, clonal haematopoiesis derived alterations can confound mutation-based assays, highlighting the need for epigenetic and multi-analyte approaches to improve specificity. Ultimately, widespread clinical adoption will require standardised, prospective trials demonstrating diagnostic accuracy and a reduction in cancer-specific mortality. Multi-analyte and machine learning-driven approaches, integrating DNA, RNA, protein and epigenomic signals, are now in late-stage clinical trials and poised for clinical translation. United States Food and Drug Administration approvals of blood-based colorectal cancer screening tests and laboratory-developed assays for hepatocellular carcinoma exemplify the translational momentum in this field. Here, we review the current landscape of liquid biopsy biomarkers for GI cancers, emphasising technological innovations, clinical performance and ongoing trials. We also discuss key challenges, including sensitivity in stage I disease, specificity amidst clonal haematopoiesis and integration with established screening paradigms. The continued evolution of assay technologies and translational research heralds a paradigm shift towards precision early detection of GI cancers, with the potential to substantially reduce mortality through earlier intervention. KEY POINTS: Liquid biopsy technologies are advancing rapidly for early detecion of GI cancers, using ctDNA, methylation profiling, fragmentomics, EVs, CTCs, and TEPs. Limited sensitivity in stage I disease remains a key barrier, largely due to low tumor burden and analyte scarcity. Clonal hematopoieses confounds mutation-based assays, emphasizing the need for epigenetic and multi-analyte strategies to improve specificity. Multi-analyte, machine-learning-driven platforms are nearing clinical translation, supported by late-stage trials and recent FDA approvals.
    Keywords:  cell‐free DNA methylation; circulating tumour DNA (ctDNA); circulating tumour cells (CTCs); early cancer detection; extracellular vesicles/exosomes; fragmentomics; gastrointestinal cancers; liquid biopsy; multi‐cancer early detection (MCED); tumour‐educated platelets (TEPs)
    DOI:  https://doi.org/10.1002/ctm2.70594
  6. Nat Med. 2026 Mar 25.
      Homologous recombination deficiency (HRD) arising from BRCA1or BRCA2 or PALB2 mutations confers sensitivity to platinum chemotherapy and PARP inhibition in pancreatic cancer (PC) and may enable prolonged disease control with immune checkpoint blockade (ICB). The phase 2 POLAR trial evaluated maintenance pembrolizumab plus olaparib following platinum-based chemotherapy in biomarker-stratified metastatic PC. Sixty-three participants were enrolled into three cohorts: cohort A (BRCA1/BRCA2-mutated or PALB2-mutated HRD, n = 33), cohort B (non-core HRD, n = 15) and cohort C (platinum sensitive, HRD-wild type, n = 15). Cohort A used a two-stage design with co-primary endpoints of at least 43% Response Evaluation Criteria in Solid Tumors (RECIST) objective response rate (ORR) and at least 77% 6-month progression-free survival (PFS) rate. Among RECIST-evaluable participants in cohort A (n = 20), ORR was 35% (95% confidence interval (CI): 15-59%), whereas 6-month PFS rate in the full cohort (n = 33) was 64% (95% CI: 49-82%), not meeting the primary endpoint. At a median follow-up of 37 months (95% CI: 27-47), median PFS and overall survival (OS) for cohort A were 8.3 (95% CI: 5.3-not reached (NR)) and 28 (95% CI: 12-NR) months, with 2-year and 3-year OS rates of 56% (95% CI: 41-76%) and 44% (95% CI: 28-69%), respectively. In cohorts B and C, ORR was 8% (95% CI: 0-38%) and 14% (95% CI: 2%-43%); median PFS was 4.8 (95% CI: 4.0-12) and 3.3 (95% CI: 1.9-4.8) months; and median OS was 18 (95% CI: 13-NR) and 10 (95% CI: 8.9-24) months, respectively. Preplanned translational analyses showed that circulating tumor DNA response, increased tumor-infiltrating lymphocytes and enrichment of frameshift indel neoantigens were associated with durable clinical benefit. These data suggest that a subset of HRD PC may derive prolonged benefit from PARP-ICB maintenance and support further development of biomarker-guided precision immunotherapy strategies in PC. ClinicalTrials.gov identifier: NCT04666740 .
    DOI:  https://doi.org/10.1038/s41591-026-04299-5
  7. Nat Genet. 2026 Mar 25.
      Sequencing the human genome came with the promise of refined risk assessment for heritable diseases, drug responses and other applications of personalized genomics. Genome-wide association studies that linked thousands of genetic alterations to heritable disorders have partially delivered on this promise. However, many patients with rare diseases remain undiagnosed after genome sequencing, in part because conventional sequencing studies struggle to characterize and phase all genomic variation. Chromosome-length phasing, enabled by the single-cell Strand-seq technique in combination with long-read data, has done much to improve the situation. For example, new diploid assembly analyses for personal genomes allow nearly complete descriptions of genomic variation. Moreover, a new Strand-seq-based phasing method can leverage DNA methylation to assign genetic variants not just to haplotypes but to maternally or paternally inherited homologous chromosomes, representing a new frontier in personalized genomics. Here we review the principles and application of Strand-seq, a key enabler of these developments.
    DOI:  https://doi.org/10.1038/s41588-026-02548-4
  8. Bioinformatics. 2026 Mar 26. pii: btag152. [Epub ahead of print]
       SUMMARY: Liquid biopsy offers a non-invasive approach to study tumor-derived genetic material circulating in plasma. Beyond genetic alterations, the fragmentomic features of cell-free DNA-such as fragment size, genomic position, and end-motifs-provide valuable insights into the biological and clinical context of DNA release. fRagmentomics is a user-friendly R package designed to characterize cfDNA fragments overlapping one or multiple small mutations of any type, starting from an aligned sequencing file (BAM). It supports multiple mutation input formats, accommodates one-based and zero-based genomic conventions, resolves mutation representation ambiguities, and accepts any reference file in FASTA format. For each fragment overlapping a mutation of interest, fRagmentomics outputs fragment-level features including its fragment size, end-motifs, and mutational status, along with additional fragment-level or read-level information. The package implements an indel-aware and optionally soft-clip-preserving fragment size computation that improves accuracy over conventional size estimates based solely on aligned positions.
    AVAILABILITY AND IMPLEMENTATION: fRagmentomics is licensed under GNU General Public License v3.0 and available at https://github.com/ElsaB-Lab/fRagmentomics and https://anaconda.org/elsab-lab/r-fragmentomics, with documentation and a tutorial.
    SUPPLEMENTARY INFORMATION: Supplementary data are available at Bioinformatics online.
    DOI:  https://doi.org/10.1093/bioinformatics/btag152
  9. PLoS One. 2026 ;21(3): e0345087
      The LINE-1 and Alu retrotransposon elements, with more than 90% of their sequences being methylated, contribute to 30% of the human genome. Their hypomethylation profile, representing global methylation in cellular and cell-free DNA (cfDNA) from cancer, has been considered an attractive noninvasive biomarker of cancer. LINE-1 and Alu methylation profiling has preferentially been performed by real-time methylation-specific PCR (qMSP), pyrosequencing, and methylation-sensitive high-resolution melting (MS-HRM), which are bisulfite-based PCR approaches that require reference sequences amplified by the Methylation Independent PCR (MIP) primers to normalize the quantification data. A technical weakness of MIP primers is unequal amplification, termed PCR amplification bias, leading to an under- or overestimation of expected methylation levels, and thus, hindering the effectiveness of DNA methylation-based biomarkers. To date, the PCR amplification bias of MIP primers that may affect the methylation analysis of repeat sequences such as LINE-1 and Alu has not yet been described. Our study demonstrated for the first time the detrimental impact of biased MIP primers on LINE-1 and Alu methylation profiles, causing a significant shift from the hypomethylated status to hypermethylated in cancer tissues and in cfDNA from cancer patients. Unexpectedly, this shift was also observed in cfDNA, even when quantified by the unbiased MIP primers, depending on the reference sequences. Our results suggest that an impartial reference for the methylation quantitation of repetitive elements, most importantly in cfDNA, should be further established to ensure cross-platform consistencies in DNA methylation profiling through bisulfite-based PCR techniques.
    DOI:  https://doi.org/10.1371/journal.pone.0345087
  10. Nat Rev Genet. 2026 Mar 23.
      Haematopoiesis has long been a paradigm for understanding how human genetic variation can influence physiology in health and disease, ranging from the genetic characterization of Mendelian blood diseases to population-scale genomic studies of blood cell phenotypes and diseases. More recently, advances in single-cell genomics and variant-to-function mapping are enabling mechanistic insights into how genetic variation shapes blood cell development. Alongside inherited variation, the characterization of somatic mutations accumulating in haematopoietic stem cells during the lifespan has revealed clonal haematopoiesis as a ubiquitous evolutionary process, with heterogeneous clonal expansions impacting haematopoietic function and disease risk. Insights from genetic studies of haematopoiesis are translating into therapeutic applications, transforming treatment for monogenic blood disorders and promising broader applications. As methods continue to advance, haematopoiesis will remain central to understanding how genetic variation influences human biology, disease susceptibility and therapeutic response.
    DOI:  https://doi.org/10.1038/s41576-026-00947-1