Targeting GM-CSF in inflammatory diseases

Nat Rev Rheumatol. 2016 Jan;12(1):37-48. doi: 10.1038/nrrheum.2015.161. Epub 2015 Dec 3.


Granulocyte-macrophage colony stimulating factor (GM-CSF) is a growth factor first identified as an inducer of differentiation and proliferation of granulocytes and macrophages derived from haematopoietic progenitor cells. Later studies have shown that GM-CSF is involved in a wide range of biological processes in both innate and adaptive immunity, with its production being tightly linked to the response to danger signals. Given that the functions of GM-CSF span multiple tissues and biological processes, this cytokine has shown potential as a new and important therapeutic target in several autoimmune and inflammatory disorders - particularly in rheumatoid arthritis. Indeed, GM-CSF was one of the first cytokines detected in human synovial fluid from inflamed joints. Therapies that target GM-CSF or its receptor have been tested in preclinical studies with promising results, further supporting the potential of targeting the GM-CSF pathway. In this Review, we discuss our expanding view of the biology of GM-CSF, outline what has been learnt about GM-CSF from studies of animal models and human diseases, and summarize the results of early phase clinical trials evaluating GM-CSF antagonism in inflammatory disorders.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Granulocyte-Macrophage Colony-Stimulating Factor / metabolism*
  • Humans
  • Immunity, Innate*
  • Immunologic Factors / therapeutic use*
  • Intercellular Signaling Peptides and Proteins / metabolism*
  • Macrophages / immunology
  • Macrophages / metabolism
  • Rheumatic Diseases* / drug therapy
  • Rheumatic Diseases* / immunology
  • Rheumatic Diseases* / metabolism
  • Synovial Fluid / metabolism


  • Immunologic Factors
  • Intercellular Signaling Peptides and Proteins
  • Granulocyte-Macrophage Colony-Stimulating Factor