TGFβ drives immune evasion in genetically reconstituted colon cancer metastasis

Nature. 2018 Feb 22;554(7693):538-543. doi: 10.1038/nature25492. Epub 2018 Feb 14.

Abstract

Most patients with colorectal cancer die as a result of the disease spreading to other organs. However, no prevalent mutations have been associated with metastatic colorectal cancers. Instead, particular features of the tumour microenvironment, such as lack of T-cell infiltration, low type 1 T-helper cell (TH1) activity and reduced immune cytotoxicity or increased TGFβ levels predict adverse outcomes in patients with colorectal cancer. Here we analyse the interplay between genetic alterations and the tumour microenvironment by crossing mice bearing conditional alleles of four main colorectal cancer mutations in intestinal stem cells. Quadruple-mutant mice developed metastatic intestinal tumours that display key hallmarks of human microsatellite-stable colorectal cancers, including low mutational burden, T-cell exclusion and TGFβ-activated stroma. Inhibition of the PD-1-PD-L1 immune checkpoint provoked a limited response in this model system. By contrast, inhibition of TGFβ unleashed a potent and enduring cytotoxic T-cell response against tumour cells that prevented metastasis. In mice with progressive liver metastatic disease, blockade of TGFβ signalling rendered tumours susceptible to anti-PD-1-PD-L1 therapy. Our data show that increased TGFβ in the tumour microenvironment represents a primary mechanism of immune evasion that promotes T-cell exclusion and blocks acquisition of the TH1-effector phenotype. Immunotherapies directed against TGFβ signalling may therefore have broad applications in treating patients with advanced colorectal cancer.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Animals
  • Cell Differentiation / drug effects
  • Colonic Neoplasms / drug therapy
  • Colonic Neoplasms / genetics*
  • Colonic Neoplasms / immunology
  • Colonic Neoplasms / pathology*
  • Disease Models, Animal
  • Drug Synergism
  • Female
  • Humans
  • Immune Evasion* / drug effects
  • Immunotherapy*
  • Intestinal Mucosa / metabolism
  • Intestines / drug effects
  • Intestines / pathology
  • Liver Neoplasms / drug therapy
  • Liver Neoplasms / immunology
  • Liver Neoplasms / secondary
  • Male
  • Mice
  • Mutation
  • Neoplasm Metastasis / drug therapy
  • Neoplasm Metastasis / genetics*
  • Neoplasm Metastasis / immunology*
  • Neoplasm Metastasis / pathology
  • Programmed Cell Death 1 Receptor / antagonists & inhibitors
  • Stem Cells / drug effects
  • Stem Cells / metabolism
  • Stem Cells / pathology
  • T-Lymphocytes, Cytotoxic / cytology
  • T-Lymphocytes, Cytotoxic / drug effects
  • T-Lymphocytes, Cytotoxic / immunology
  • Th1 Cells / drug effects
  • Th1 Cells / immunology
  • Transforming Growth Factor beta / antagonists & inhibitors
  • Transforming Growth Factor beta / immunology*
  • Tumor Microenvironment / drug effects
  • Tumor Microenvironment / immunology

Substances

  • Programmed Cell Death 1 Receptor
  • Transforming Growth Factor beta