CD1d-antibody fusion proteins target iNKT cells to the tumor and trigger long-term therapeutic responses

Cancer Immunol Immunother. 2013 Apr;62(4):747-60. doi: 10.1007/s00262-012-1381-7. Epub 2012 Dec 15.

Abstract

Despite the well-established antitumor activity of CD1d-restricted invariant natural killer T lymphocytes (iNKT), their use for cancer therapy has remained challenging. This appears to be due to their strong but short-lived activation followed by long-term anergy after a single administration of the CD1d agonist ligand alpha-galactosylceramide (αGC). As a promising alternative, we obtained sustained mouse iNKT cell responses associated with prolonged antitumor effects through repeated administrations of tumor-targeted recombinant sCD1d-antitumor scFv fusion proteins loaded with αGC. Here, we demonstrate that CD1d fusion proteins bound to tumor cells via the antibody fragment specific for a tumor-associated antigen, efficiently activate human iNKT cell lines leading to potent tumor cell lysis. The importance of CD1d tumor targeting was confirmed in tumor-bearing mice in which only the specific tumor-targeted CD1d fusion protein resulted in tumor inhibition of well-established aggressive tumor grafts. The therapeutic efficacy correlated with the repeated activation of iNKT and natural killer cells marked by their release of TH1 cytokines, despite the up-regulation of the co-inhibitory receptor PD-1. Our results demonstrate the superiority of providing the superagonist αGC loaded on recombinant CD1d proteins and support the use of αGC/sCD1d-antitumor fusion proteins to secure a sustained human and mouse iNKT cell activation, while targeting their cytotoxic activity and cytokine release to the tumor site.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antigens, CD1d / immunology
  • Antigens, CD1d / pharmacology*
  • Cell Line, Tumor
  • Female
  • Galactosylceramides / immunology
  • Humans
  • Immunoglobulin Fragments / immunology
  • Immunoglobulin Fragments / pharmacology*
  • Immunotherapy, Adoptive / methods*
  • Killer Cells, Natural / drug effects
  • Killer Cells, Natural / immunology
  • Mice
  • Mice, Inbred C57BL
  • Natural Killer T-Cells / drug effects*
  • Natural Killer T-Cells / immunology*
  • Recombinant Fusion Proteins / immunology
  • Recombinant Fusion Proteins / pharmacology*

Substances

  • Antigens, CD1d
  • CD1D protein, human
  • Galactosylceramides
  • Immunoglobulin Fragments
  • Recombinant Fusion Proteins
  • alpha-galactosylceramide