Acetylation-dependent regulation of PD-L1 nuclear translocation dictates the efficacy of anti-PD-1 immunotherapy

Nat Cell Biol. 2020 Sep;22(9):1064-1075. doi: 10.1038/s41556-020-0562-4. Epub 2020 Aug 24.

Abstract

Immunotherapies that target programmed cell death protein 1 (PD-1) and its ligand PD-L1 as well as cytotoxic T-lymphocyte-associated protein 4 (CTLA4) have shown impressive clinical outcomes for multiple tumours. However, only a subset of patients achieves durable responses, suggesting that the mechanisms of the immune checkpoint pathways are not completely understood. Here, we report that PD-L1 translocates from the plasma membrane into the nucleus through interactions with components of the endocytosis and nucleocytoplasmic transport pathways, regulated by p300-mediated acetylation and HDAC2-dependent deacetylation of PD-L1. Moreover, PD-L1 deficiency leads to compromised expression of multiple immune-response-related genes. Genetically or pharmacologically modulating PD-L1 acetylation blocks its nuclear translocation, reprograms the expression of immune-response-related genes and, as a consequence, enhances the anti-tumour response to PD-1 blockade. Thus, our results reveal an acetylation-dependent regulation of PD-L1 nuclear localization that governs immune-response gene expression, and thereby advocate targeting PD-L1 translocation to enhance the efficacy of PD-1/PD-L1 blockade.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylation
  • Animals
  • B7-H1 Antigen / metabolism*
  • Cell Line
  • Cell Line, Tumor
  • Cell Nucleus / metabolism*
  • E1A-Associated p300 Protein / metabolism
  • Gene Expression / physiology
  • HEK293 Cells
  • Humans
  • Immunotherapy / methods
  • MCF-7 Cells
  • Mice
  • Neoplasms / metabolism
  • Programmed Cell Death 1 Receptor / metabolism*
  • Protein Processing, Post-Translational / physiology
  • RAW 264.7 Cells

Substances

  • B7-H1 Antigen
  • CD274 protein, human
  • PDCD1 protein, human
  • Programmed Cell Death 1 Receptor
  • E1A-Associated p300 Protein