Loss of IFN-γ Pathway Genes in Tumor Cells as a Mechanism of Resistance to Anti-CTLA-4 Therapy

Cell. 2016 Oct 6;167(2):397-404.e9. doi: 10.1016/j.cell.2016.08.069. Epub 2016 Sep 22.

Abstract

Antibody blockade of the inhibitory CTLA-4 pathway has led to clinical benefit in a subset of patients with metastatic melanoma. Anti-CTLA-4 enhances T cell responses, including production of IFN-γ, which is a critical cytokine for host immune responses. However, the role of IFN-γ signaling in tumor cells in the setting of anti-CTLA-4 therapy remains unknown. Here, we demonstrate that patients identified as non-responders to anti-CTLA-4 (ipilimumab) have tumors with genomic defects in IFN-γ pathway genes. Furthermore, mice bearing melanoma tumors with knockdown of IFN-γ receptor 1 (IFNGR1) have impaired tumor rejection upon anti-CTLA-4 therapy. These data highlight that loss of the IFN-γ signaling pathway is associated with primary resistance to anti-CTLA-4 therapy. Our findings demonstrate the importance of tumor genomic data, especially IFN-γ related genes, as prognostic information for patients selected to receive treatment with immune checkpoint therapy.

Keywords: IFN-γ signaling; Melanoma; anti-CTLA-4; copy-number alteration; ipilimumab; primary resistance.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Antibodies, Monoclonal / therapeutic use*
  • CTLA-4 Antigen / antagonists & inhibitors*
  • Cell Line, Tumor
  • Cytokines / immunology
  • Drug Resistance, Neoplasm / genetics*
  • Gene Knockdown Techniques
  • Humans
  • Interferon gamma Receptor
  • Interferon-gamma / genetics*
  • Ipilimumab
  • Melanoma / drug therapy*
  • Melanoma / genetics
  • Melanoma, Experimental / drug therapy
  • Melanoma, Experimental / genetics
  • Mice
  • Mice, Inbred C57BL
  • Receptors, Interferon / genetics*
  • Skin Neoplasms / drug therapy*
  • Skin Neoplasms / genetics
  • T-Lymphocytes / immunology

Substances

  • Antibodies, Monoclonal
  • CTLA-4 Antigen
  • CTLA4 protein, human
  • Cytokines
  • Ipilimumab
  • Receptors, Interferon
  • Interferon-gamma