alpha-Galactosylceramide therapy for autoimmune diseases: prospects and obstacles

Nat Rev Immunol. 2005 Jan;5(1):31-42. doi: 10.1038/nri1531.


Autoimmune responses are normally kept in check by immune-tolerance mechanisms, which include regulatory T cells. In recent years, research has focused on the role of a subset of natural killer T (NKT) cells - invariant NKT (iNKT) cells, which are a population of glycolipid-reactive regulatory T cells - in controlling autoimmune responses. Because iNKT cells strongly react with a marine-sponge-derived glycolipid, alpha-galactosylceramide (alpha-GalCer), it has been possible to specifically target and track these cells. As I discuss here, although preclinical studies have shown considerable promise for the development of treatment with alpha-GalCer as a therapeutic modality for autoimmune diseases, several obstacles need to be overcome before moving alpha-GalCer therapy from the bench to the bedside.

Publication types

  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Autoimmune Diseases / drug therapy*
  • Autoimmune Diseases / immunology
  • Autoimmunity / drug effects*
  • Autoimmunity / immunology
  • Galactosylceramides / pharmacology*
  • Galactosylceramides / therapeutic use
  • Humans
  • Immunologic Factors / pharmacology*
  • Killer Cells, Natural / drug effects
  • Killer Cells, Natural / immunology
  • T-Lymphocyte Subsets / drug effects
  • T-Lymphocyte Subsets / immunology


  • Galactosylceramides
  • Immunologic Factors
  • alpha-galactosylceramide