Small molecule mediated inhibition of RORγ-dependent gene expression and autoimmune disease pathology in vivo

Immunology. 2016 Apr;147(4):399-413. doi: 10.1111/imm.12570. Epub 2016 Jan 26.

Abstract

Retinoic acid receptor-related orphan nuclear receptor γ (RORγ) orchestrates a pro-inflammatory gene expression programme in multiple lymphocyte lineages including T helper type 17 (Th17) cells, γδ T cells, innate lymphoid cells and lymphoid tissue inducer cells. There is compelling evidence that RORγ-expressing cells are relevant targets for therapeutic intervention in the treatment of autoimmune and inflammatory diseases. Unlike Th17 cells, where RORγ expression is induced under specific pro-inflammatory conditions, γδ T cells and other innate-like immune cells express RORγ in the steady state. Small molecule mediated disruption of RORγ function in cells with pre-existing RORγ transcriptional complexes represents a significant and challenging pharmacological hurdle. We present data demonstrating that a novel, selective and potent small molecule RORγ inhibitor can block the RORγ-dependent gene expression programme in both Th17 cells and RORγ-expressing γδ T cells as well as a disease-relevant subset of human RORγ-expressing memory T cells. Importantly, systemic administration of this inhibitor in vivo limits pathology in an innate lymphocyte-driven mouse model of psoriasis.

Keywords: ROR γ; T helper type 17; imiquimod; γδ T cells and autoimmunity.

MeSH terms

  • Animals
  • Autoimmune Diseases / etiology*
  • Autoimmune Diseases / metabolism*
  • Benzamides / pharmacology*
  • Cell Differentiation / drug effects
  • Cell Differentiation / immunology
  • Dermatitis / drug therapy
  • Dermatitis / immunology
  • Dermatitis / metabolism
  • Dermatitis / pathology
  • Disease Models, Animal
  • Dose-Response Relationship, Drug
  • Gene Expression Regulation / drug effects*
  • Humans
  • Immunologic Memory / drug effects
  • Interleukin-17 / metabolism
  • Mice
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / antagonists & inhibitors
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / metabolism*
  • Pyridines / pharmacology*
  • Receptors, Antigen, T-Cell, gamma-delta / metabolism
  • T-Lymphocyte Subsets / cytology
  • T-Lymphocyte Subsets / immunology
  • T-Lymphocyte Subsets / metabolism
  • T-Lymphocytes, Helper-Inducer / cytology
  • T-Lymphocytes, Helper-Inducer / immunology
  • T-Lymphocytes, Helper-Inducer / metabolism
  • Th17 Cells / cytology
  • Th17 Cells / immunology
  • Th17 Cells / metabolism

Substances

  • BIO-0554019
  • Benzamides
  • Interleukin-17
  • Nuclear Receptor Subfamily 1, Group F, Member 3
  • Pyridines
  • Receptors, Antigen, T-Cell, gamma-delta