A small-molecule c-Rel inhibitor reduces alloactivation of T cells without compromising antitumor activity

Cancer Discov. 2014 May;4(5):578-91. doi: 10.1158/2159-8290.CD-13-0585. Epub 2014 Feb 18.

Abstract

Preventing unfavorable GVHD without inducing broad suppression of the immune system presents a major challenge of allogeneic hematopoietic stem cell transplantation (allo-HSCT). We developed a novel strategy to ameliorate GVHD while preserving graft-versus-tumor (GVT) activity by small molecule-based inhibition of the NF-κB family member c-Rel. Underlying mechanisms included reduced alloactivation, defective gut homing, and impaired negative feedback on interleukin (IL)-2 production, resulting in optimal IL-2 levels, which, in the absence of competition by effector T cells, translated into expansion of regulatory T cells. c-Rel activity was dispensable for antigen-specific T-cell receptor (TCR) activation, allowing c-Rel-deficient T cells to display normal GVT activity. In addition, inhibition of c-Rel activity reduced alloactivation without compromising antigen-specific cytotoxicity of human T cells. Finally, we were able to demonstrate the feasibility and efficacy of systemic c-Rel inhibitor administration. Our findings validate c-Rel as a promising target for immunomodulatory therapy and demonstrate the feasibility and efficacy of pharmaceutical inhibition of c-Rel activity.

Publication types

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

MeSH terms

  • Animals
  • Female
  • Gene Expression Regulation
  • Graft vs Host Disease / immunology
  • Graft vs Host Disease / prevention & control*
  • Graft vs Tumor Effect / immunology
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Humans
  • Lymphocyte Activation / drug effects*
  • Lymphocyte Activation / immunology
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred C57BL
  • Proto-Oncogene Proteins c-rel / antagonists & inhibitors*
  • Proto-Oncogene Proteins c-rel / genetics
  • Proto-Oncogene Proteins c-rel / metabolism
  • Receptors, Antigen, T-Cell / metabolism
  • Small Molecule Libraries / pharmacology*
  • T-Lymphocytes / drug effects*
  • T-Lymphocytes / immunology
  • Transplantation, Homologous

Substances

  • Proto-Oncogene Proteins c-rel
  • Receptors, Antigen, T-Cell
  • Small Molecule Libraries