Targeting CXCL12 from FAP-expressing carcinoma-associated fibroblasts synergizes with anti-PD-L1 immunotherapy in pancreatic cancer

Proc Natl Acad Sci U S A. 2013 Dec 10;110(50):20212-7. doi: 10.1073/pnas.1320318110. Epub 2013 Nov 25.

Abstract

An autochthonous model of pancreatic ductal adenocarcinoma (PDA) permitted the analysis of why immunotherapy is ineffective in this human disease. Despite finding that PDA-bearing mice had cancer cell-specific CD8(+) T cells, the mice, like human patients with PDA, did not respond to two immunological checkpoint antagonists that promote the function of T cells: anti-cytotoxic T-lymphocyte-associated protein 4 (α-CTLA-4) and α-programmed cell death 1 ligand 1 (α-PD-L1). Immune control of PDA growth was achieved, however, by depleting carcinoma-associated fibroblasts (CAFs) that express fibroblast activation protein (FAP). The depletion of the FAP(+) stromal cell also uncovered the antitumor effects of α-CTLA-4 and α-PD-L1, indicating that its immune suppressive activity accounts for the failure of these T-cell checkpoint antagonists. Three findings suggested that chemokine (C-X-C motif) ligand 12 (CXCL12) explained the overriding immunosuppression by the FAP(+) cell: T cells were absent from regions of the tumor containing cancer cells, cancer cells were coated with the chemokine, CXCL12, and the FAP(+) CAF was the principal source of CXCL12 in the tumor. Administering AMD3100, a CXCL12 receptor chemokine (C-X-C motif) receptor 4 inhibitor, induced rapid T-cell accumulation among cancer cells and acted synergistically with α-PD-L1 to greatly diminish cancer cells, which were identified by their loss of heterozygosity of Trp53 gene. The residual tumor was composed only of premalignant epithelial cells and inflammatory cells. Thus, a single protein, CXCL12, from a single stromal cell type, the FAP(+) CAF, may direct tumor immune evasion in a model of human PDA.

Keywords: KPC mouse; T cell exclusion; tumor immunogenicity; tumor stroma.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Analysis of Variance
  • Animals
  • Base Sequence
  • Benzylamines
  • Carcinoma, Pancreatic Ductal / immunology
  • Carcinoma, Pancreatic Ductal / therapy*
  • Chemokine CXCL12 / metabolism*
  • Cyclams
  • Endopeptidases
  • Enzyme-Linked Immunospot Assay
  • Fibroblasts / metabolism
  • Flow Cytometry
  • Fluorescent Antibody Technique
  • Gelatinases / metabolism*
  • Heterocyclic Compounds
  • Immunohistochemistry
  • Immunotherapy / methods*
  • Membrane Proteins / metabolism*
  • Mice
  • Molecular Sequence Data
  • Pancreatic Neoplasms / immunology
  • Pancreatic Neoplasms / therapy*
  • Sequence Analysis, RNA
  • Serine Endopeptidases / metabolism*
  • Tumor Escape / genetics*

Substances

  • Benzylamines
  • Chemokine CXCL12
  • Cxcl12 protein, mouse
  • Cyclams
  • Heterocyclic Compounds
  • Membrane Proteins
  • Endopeptidases
  • Serine Endopeptidases
  • fibroblast activation protein alpha
  • Gelatinases
  • plerixafor

Associated data

  • GEO/GSE42605