Specific Btk inhibition suppresses B cell- and myeloid cell-mediated arthritis

Nat Chem Biol. 2011 Jan;7(1):41-50. doi: 10.1038/nchembio.481. Epub 2010 Nov 28.

Abstract

Bruton's tyrosine kinase (Btk) is a therapeutic target for rheumatoid arthritis, but the cellular and molecular mechanisms by which Btk mediates inflammation are poorly understood. Here we describe the discovery of CGI1746, a small-molecule Btk inhibitor chemotype with a new binding mode that stabilizes an inactive nonphosphorylated enzyme conformation. CGI1746 has exquisite selectivity for Btk and inhibits both auto- and transphosphorylation steps necessary for enzyme activation. Using CGI1746, we demonstrate that Btk regulates inflammatory arthritis by two distinct mechanisms. CGI1746 blocks B cell receptor-dependent B cell proliferation and in prophylactic regimens reduces autoantibody levels in collagen-induced arthritis. In macrophages, Btk inhibition abolishes FcγRIII-induced TNFα, IL-1β and IL-6 production. Accordingly, in myeloid- and FcγR-dependent autoantibody-induced arthritis, CGI1746 decreases cytokine levels within joints and ameliorates disease. These results provide new understanding of the function of Btk in both B cell- or myeloid cell-driven disease processes and provide a compelling rationale for targeting Btk in rheumatoid arthritis.

MeSH terms

  • Agammaglobulinaemia Tyrosine Kinase
  • Animals
  • Arthritis, Experimental / drug therapy*
  • Arthritis, Experimental / immunology
  • Arthritis, Experimental / metabolism
  • Arthritis, Rheumatoid / drug therapy*
  • Arthritis, Rheumatoid / immunology
  • Arthritis, Rheumatoid / metabolism
  • Autoantibodies / immunology
  • Autoantibodies / metabolism
  • B-Lymphocytes / drug effects*
  • B-Lymphocytes / immunology
  • B-Lymphocytes / metabolism
  • Benzamides / chemistry
  • Benzamides / pharmacology
  • Benzamides / therapeutic use*
  • Bridged Bicyclo Compounds, Heterocyclic / chemistry
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use*
  • Cell Proliferation / drug effects
  • Enzyme Activation / drug effects
  • Interleukin-1beta / immunology
  • Interleukin-1beta / metabolism
  • Interleukin-6 / immunology
  • Interleukin-6 / metabolism
  • Mice
  • Myeloid Cells / drug effects*
  • Myeloid Cells / immunology
  • Myeloid Cells / metabolism
  • Phosphorylation / drug effects
  • Protein Kinase Inhibitors / chemistry
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use*
  • Protein-Tyrosine Kinases / chemistry
  • Protein-Tyrosine Kinases / pharmacology
  • Protein-Tyrosine Kinases / therapeutic use
  • Tumor Necrosis Factor-alpha / immunology
  • Tumor Necrosis Factor-alpha / metabolism

Substances

  • Autoantibodies
  • Benzamides
  • Bridged Bicyclo Compounds, Heterocyclic
  • CGI 1746
  • Interleukin-1beta
  • Interleukin-6
  • Protein Kinase Inhibitors
  • Tumor Necrosis Factor-alpha
  • Protein-Tyrosine Kinases
  • Agammaglobulinaemia Tyrosine Kinase
  • Btk protein, mouse

Associated data

  • PDB/3OCS
  • PDB/3OCT
  • PDB/3P08
  • PubChem-Substance/99453934
  • PubChem-Substance/99453935
  • PubChem-Substance/99453936
  • PubChem-Substance/99453937
  • PubChem-Substance/99453938
  • PubChem-Substance/99453939
  • PubChem-Substance/99453940