CS1-specific chimeric antigen receptor (CAR)-engineered natural killer cells enhance in vitro and in vivo antitumor activity against human multiple myeloma

Leukemia. 2014 Apr;28(4):917-27. doi: 10.1038/leu.2013.279. Epub 2013 Sep 26.

Abstract

Multiple myeloma (MM) is an incurable hematological malignancy. Chimeric antigen receptor (CAR)-expressing T cells have been demonstrated successfully in the clinic to treat B-lymphoid malignancies. However, the potential utility of antigen-specific CAR-engineered natural-killer (NK) cells to treat MM has not been explored. In this study, we determined whether CS1, a surface protein that is highly expressed on MM cells, can be targeted by CAR NK cells to treat MM. We successfully generated a viral construct of a CS1-specific CAR and expressed it in human NK cells. In vitro, CS1-CAR NK cells displayed enhanced MM cytolysis and interferon-γ (IFN-γ) production, and showed a specific CS1-dependent recognition of MM cells. Ex vivo, CS1-CAR NK cells also showed similarly enhanced activities when responding to primary MM tumor cells. More importantly, in an aggressive orthotopic MM xenograft mouse model, adoptive transfer of NK-92 cells expressing CS1-CAR efficiently suppressed the growth of human IM9 MM cells and also significantly prolonged mouse survival. Thus, CS1 represents a viable target for CAR-expressing immune cells, and autologous or allogeneic transplantation of CS1-specific CAR NK cells may be a promising strategy to treat MM.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cells, Cultured
  • Cytotoxicity, Immunologic
  • Genetic Engineering
  • Humans
  • Interferon-gamma / biosynthesis
  • Killer Cells, Natural / immunology*
  • Killer Cells, Natural / metabolism
  • Lentivirus / genetics
  • Mice
  • Mice, Inbred NOD
  • Multiple Myeloma / mortality
  • Multiple Myeloma / therapy*
  • Phenotype
  • Receptors, Antigen / genetics*
  • Receptors, Immunologic / immunology*
  • Signaling Lymphocytic Activation Molecule Family
  • Xenograft Model Antitumor Assays

Substances

  • Receptors, Antigen
  • Receptors, Immunologic
  • SLAMF7 protein, human
  • Signaling Lymphocytic Activation Molecule Family
  • Interferon-gamma