Small Molecule Enhancers of Endosome-to-Cytosol Import Augment Anti-tumor Immunity

Cell Rep. 2020 Jul 14;32(2):107905. doi: 10.1016/j.celrep.2020.107905.

Abstract

Cross-presentation of antigens by dendritic cells (DCs) is critical for initiation of anti-tumor immune responses. Yet, key steps involved in trafficking of antigens taken up by DCs remain incompletely understood. Here, we screen 700 US Food and Drug Administration (FDA)-approved drugs and identify 37 enhancers of antigen import from endolysosomes into the cytosol. To reveal their mechanism of action, we generate proteomic organellar maps of control and drug-treated DCs (focusing on two compounds, prazosin and tamoxifen). By combining organellar mapping, quantitative proteomics, and microscopy, we conclude that import enhancers undergo lysosomal trapping leading to membrane permeation and antigen release. Enhancing antigen import facilitates cross-presentation of soluble and cell-associated antigens. Systemic administration of prazosin leads to reduced growth of MC38 tumors and to a synergistic effect with checkpoint immunotherapy in a melanoma model. Thus, inefficient antigen import into the cytosol limits antigen cross-presentation, restraining the potency of anti-tumor immune responses and efficacy of checkpoint blockers.

Keywords: cross-presentation; dendritic cells; dynamic organellar maps; immunotherapy; lysosomes; organellar proteomics; small molecule screening.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antigens / metabolism
  • Antineoplastic Agents / pharmacology*
  • Biological Transport / drug effects
  • Cross-Priming / drug effects
  • Cytosol / drug effects
  • Cytosol / metabolism*
  • Dendritic Cells / metabolism
  • Endoplasmic Reticulum-Associated Degradation / drug effects
  • Endosomes / drug effects
  • Endosomes / metabolism*
  • Immunity* / drug effects
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Neoplasms / drug therapy
  • Neoplasms / immunology*
  • Permeability
  • Prazosin / pharmacology
  • Quinazolines / pharmacology
  • Small Molecule Libraries / pharmacology*
  • Tamoxifen / pharmacology
  • beta-Lactamases / metabolism

Substances

  • Antigens
  • Antineoplastic Agents
  • Quinazolines
  • Small Molecule Libraries
  • Tamoxifen
  • beta-Lactamases
  • Prazosin