Btk-specific inhibition blocks pathogenic plasma cell signatures and myeloid cell-associated damage in IFN α-driven lupus nephritis

JCI Insight. 2017 Apr 6;2(7):e90111. doi: 10.1172/jci.insight.90111.

Abstract

Systemic lupus erythematosus (SLE) is often associated with exaggerated B cell activation promoting plasma cell generation, immune-complex deposition in the kidney, renal infiltration of myeloid cells, and glomerular nephritis. Type-I IFNs amplify these autoimmune processes and promote severe disease. Bruton's tyrosine kinase (Btk) inhibitors are considered novel therapies for SLE. We describe the characterization of a highly selective reversible Btk inhibitor, G-744. G-744 is efficacious, and superior to blocking BAFF and Syk, in ameliorating severe lupus nephritis in both spontaneous and IFNα-accelerated lupus in NZB/W_F1 mice in therapeutic regimens. Selective Btk inhibition ablated plasmablast generation, reduced autoantibodies, and - similar to cyclophosphamide - improved renal pathology in IFNα-accelerated lupus. Employing global transcriptional profiling of spleen and kidney coupled with cross-species human modular repertoire analyses, we identify similarities in the inflammatory process between mice and humans, and we demonstrate that G-744 reduced gene expression signatures essential for splenic B cell terminal differentiation, particularly the secretory pathway, as well as renal transcriptional profiles coupled with myeloid cell-mediated pathology and glomerular plus tubulointerstitial disease in human glomerulonephritis patients. These findings reveal the mechanism through which a selective Btk inhibitor blocks murine autoimmune kidney disease, highlighting pathway activity that may translate to human SLE.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Agammaglobulinaemia Tyrosine Kinase / antagonists & inhibitors*
  • Agammaglobulinaemia Tyrosine Kinase / metabolism
  • Animals
  • Autoantibodies / immunology
  • B-Lymphocytes / drug effects
  • B-Lymphocytes / immunology*
  • Cell Proliferation / drug effects
  • Disease Models, Animal
  • Female
  • Gene Expression / drug effects
  • Humans
  • Interferon-alpha / immunology
  • Kidney / immunology
  • Kidney / pathology
  • Lupus Nephritis / immunology*
  • Lupus Nephritis / metabolism
  • Lymphocyte Activation / drug effects
  • Mice
  • Mice, Inbred NZB
  • Myeloid Cells / metabolism*
  • Plasma Cells / drug effects
  • Plasma Cells / pathology*

Substances

  • Autoantibodies
  • Interferon-alpha
  • Agammaglobulinaemia Tyrosine Kinase
  • Btk protein, mouse