Clinical experience with α-galactosylceramide (KRN7000) in patients with advanced cancer and chronic hepatitis B/C infection

Clin Immunol. 2011 Aug;140(2):130-41. doi: 10.1016/j.clim.2010.11.010. Epub 2010 Dec 18.


For over a century, research has sought ways to boost the immune system in order to eradicate tumors and viruses that exist after escaping immunosurveillance. For the treatment of cancer and hepatitis immunotherapeutic strategies have overall had limited clinical success. An urgent need exists therefore to introduce more effective therapeutic approaches. Invariant (i)NKT cells constitute a conserved T lymphocyte lineage with dominant immunoregulatory, antitumor and antiviral effector cell properties. iNKT specifically recognize the glycolipid α-galactosylceramide in the context of CD1d resulting in their activation. Activated iNKT can promote the development of a long-lasting Th1 biased proinflammatory immune response as demonstrated in multiple tumor-metastasis and viral infection models. Here, we will provide a brief overview of the preclinical data of α-galactosylceramide that formed the basis for subsequent clinical trials in patients with advanced cancer and chronic hepatitis B/C, and elaborate on our own clinical experience with α-galactosylceramide in these patient groups.

Publication types

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

MeSH terms

  • Antigens, CD1d / immunology
  • Antineoplastic Agents / immunology
  • Antineoplastic Agents / therapeutic use
  • Clinical Trials as Topic
  • Galactosylceramides / immunology*
  • Galactosylceramides / therapeutic use
  • Hepatitis B, Chronic / drug therapy
  • Hepatitis B, Chronic / immunology*
  • Hepatitis C, Chronic / drug therapy
  • Hepatitis C, Chronic / immunology*
  • Humans
  • Killer Cells, Natural / immunology
  • Lymphocyte Activation / drug effects
  • Lymphocyte Activation / immunology
  • Neoplasms / drug therapy
  • Neoplasms / immunology*


  • Antigens, CD1d
  • Antineoplastic Agents
  • Galactosylceramides
  • KRN 7000