Identification of an allosteric binding site for RORγt inhibition

Nat Commun. 2015 Dec 7:6:8833. doi: 10.1038/ncomms9833.

Abstract

RORγt is critical for the differentiation and proliferation of Th17 cells associated with several chronic autoimmune diseases. We report the discovery of a novel allosteric binding site on the nuclear receptor RORγt. Co-crystallization of the ligand binding domain (LBD) of RORγt with a series of small-molecule antagonists demonstrates occupancy of a previously unreported allosteric binding pocket. Binding at this non-canonical site induces an unprecedented conformational reorientation of helix 12 in the RORγt LBD, which blocks cofactor binding. The functional consequence of this allosteric ligand-mediated conformation is inhibition of function as evidenced by both biochemical and cellular studies. RORγt function is thus antagonized in a manner molecularly distinct from that of previously described orthosteric RORγt ligands. This brings forward an approach to target RORγt for the treatment of Th17-mediated autoimmune diseases. The elucidation of an unprecedented modality of pharmacological antagonism establishes a mechanism for modulation of nuclear receptors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Allosteric Site
  • Animals
  • Cell Differentiation
  • Humans
  • Interleukin-17 / chemistry
  • Interleukin-17 / metabolism*
  • Ligands
  • Mice
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / chemistry*
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / genetics
  • Nuclear Receptor Subfamily 1, Group F, Member 3 / metabolism*
  • Protein Structure, Tertiary
  • Th17 Cells / chemistry
  • Th17 Cells / metabolism

Substances

  • Interleukin-17
  • Ligands
  • Nuclear Receptor Subfamily 1, Group F, Member 3
  • RORC protein, human
  • Rorc protein, mouse