Spatial Heterogeneity, Stromal Phenotypes, and Therapeutic Vulnerabilities in Colorectal Cancer Peritoneal Metastasis

Clin Cancer Res. 2025 Jun 13;31(12):2515-2529. doi: 10.1158/1078-0432.CCR-24-3780.

Abstract

Purpose: Peritoneal metastases (PM) in colorectal cancer portend a poor prognosis. We sought to elucidate molecular features differentiating primary tumors (PT) from PMs and actionable targets facilitating transcoelomic dissemination and progression.

Experimental design: We performed multiomic profiling of 227 samples from 136 patients, including 56 PTs and 120 synchronous PMs comprising 34 matched PT-PM pairs. Whole-exome and bulk RNA sequencing analyses were conducted to identify underlying genomic aberrations and transcriptomic differences between primary and peritoneal lesions. We spatially characterized the microenvironment of tumor-stroma compartments and studied the roles of stromal phenotypes in promulgating tumorigenesis.

Results: Whole-exome sequencing found that genomic alterations and clonality patterns between PTs and PMs remain broadly similar. Transcriptomic profiles, however, suggest a transition as tumors reach the peritoneum toward a more mesenchymal tumor profile and fibrotic tumor microenvironment. Applying spatial profiling, we identify a fibro-collagenous and immune-infiltrated stromal phenotype [stromal cluster (SC) 2] characterized by increased cancer-associated fibroblasts, memory B cells, M2 macrophages, and T-cell exhaustion. These findings were orthogonally validated by multiplex IHC. Patients with SC2 stroma had poorer survival and were characterized by high SERPINE-1 (PAI-1) expression. PMs in patients with SC2 stroma were associated with enriched oncogenic pathways such as TGF-β. PAI-1 inhibition of colorectal cancer PM cell lines with a novel biologic demonstrated reduced IL2-STAT5 and TGF-β pathways and cell death.

Conclusions: Our findings unveil distinctive and actionable molecular signatures, offering deeper insights into the intricate cross-talk between tumor cells and stromal microenvironments enabling PM in colorectal cancer.

MeSH terms

  • Aged
  • Biomarkers, Tumor* / genetics
  • Cancer-Associated Fibroblasts / metabolism
  • Cancer-Associated Fibroblasts / pathology
  • Colorectal Neoplasms* / genetics
  • Colorectal Neoplasms* / pathology
  • Exome Sequencing
  • Female
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Male
  • Middle Aged
  • Peritoneal Neoplasms* / genetics
  • Peritoneal Neoplasms* / secondary
  • Peritoneal Neoplasms* / therapy
  • Phenotype
  • Prognosis
  • Stromal Cells* / metabolism
  • Stromal Cells* / pathology
  • Transcriptome
  • Tumor Microenvironment / genetics
  • Tumor Microenvironment / immunology

Substances

  • Biomarkers, Tumor