Discovery of Small Molecules for the Reversal of T Cell Exhaustion

Cell Rep. 2019 Dec 3;29(10):3293-3302.e3. doi: 10.1016/j.celrep.2019.10.119.

Abstract

Inhibitory receptors (IRs) function as critical regulators of immune responses by tempering T cell activity. In humans, several persisting viruses as well as cancers exploit IR signaling by upregulating IR ligands, resulting in suppression of T cell function (i.e., exhaustion). This allows escape from immune surveillance and continuation of disease. Here, we report the design, implementation, and results of a phenotypic high-throughput screen for molecules that modulate CD8+ T cell activity. We identify 19 compounds from the ReFRAME drug-repurposing collection that restore cytokine production and enhance the proliferation of exhausted T cells. Analysis of our top hit, ingenol mebutate, a protein kinase C (PKC) inducing diterpene ester, reveals a role for this molecule in overriding the suppressive signaling cascade mediated by IR signaling on T cells. Collectively, these results demonstrate a disease-relevant methodology for identifying modulators of T cell function and reveal new targets for immunotherapy.

Keywords: CD8 T cell; PKC; T cell exhaustion; checkpoint blockade; chronic infection; high-throughput flow cytometry; ingenol mebutate.

Publication types

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

MeSH terms

  • Animals
  • CD8-Positive T-Lymphocytes / drug effects*
  • CD8-Positive T-Lymphocytes / metabolism
  • Cell Proliferation / drug effects
  • Chlorocebus aethiops
  • Costimulatory and Inhibitory T-Cell Receptors / metabolism
  • Cytokines / metabolism
  • Immunotherapy / methods
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Protein Kinase C / metabolism
  • Signal Transduction / drug effects
  • Small Molecule Libraries / pharmacology*
  • Vero Cells

Substances

  • Costimulatory and Inhibitory T-Cell Receptors
  • Cytokines
  • Small Molecule Libraries
  • Protein Kinase C