Selective inhibition of tumor growth by clonal NK cells expressing an ErbB2/HER2-specific chimeric antigen receptor

Mol Ther. 2015 Feb;23(2):330-8. doi: 10.1038/mt.2014.219. Epub 2014 Nov 6.

Abstract

Natural killer (NK) cells are an important effector cell type for adoptive cancer immunotherapy. Similar to T cells, NK cells can be modified to express chimeric antigen receptors (CARs) to enhance antitumor activity, but experience with CAR-engineered NK cells and their clinical development is still limited. Here, we redirected continuously expanding and clinically usable established human NK-92 cells to the tumor-associated ErbB2 (HER2) antigen. Following GMP-compliant procedures, we generated a stable clonal cell line expressing a humanized CAR based on ErbB2-specific antibody FRP5 harboring CD28 and CD3ζ signaling domains (CAR 5.28.z). These NK-92/5.28.z cells efficiently lysed ErbB2-expressing tumor cells in vitro and exhibited serial target cell killing. Specific recognition of tumor cells and antitumor activity were retained in vivo, resulting in selective enrichment of NK-92/5.28.z cells in orthotopic breast carcinoma xenografts, and reduction of pulmonary metastasis in a renal cell carcinoma model, respectively. γ-irradiation as a potential safety measure for clinical application prevented NK cell replication, while antitumor activity was preserved. Our data demonstrate that it is feasible to engineer CAR-expressing NK cells as a clonal, molecularly and functionally well-defined and continuously expandable cell therapeutic agent, and suggest NK-92/5.28.z cells as a promising candidate for use in adoptive cancer immunotherapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Breast Neoplasms / genetics
  • Breast Neoplasms / immunology
  • Breast Neoplasms / therapy
  • Cell Line, Transformed
  • Cell Line, Tumor
  • Clonal Evolution
  • Cytotoxicity, Immunologic
  • Disease Models, Animal
  • Epitopes, T-Lymphocyte / immunology
  • Female
  • Gene Expression*
  • Genetic Vectors / genetics
  • Humans
  • Immunophenotyping
  • Immunotherapy
  • Killer Cells, Natural / immunology*
  • Killer Cells, Natural / metabolism*
  • Lentivirus / genetics
  • Lymphocyte Culture Test, Mixed
  • Neoplasms / genetics
  • Neoplasms / immunology
  • Neoplasms / pathology
  • Neoplasms / therapy
  • Phenotype
  • Receptor, ErbB-2 / immunology*
  • Receptors, Immunologic / genetics*
  • Receptors, Immunologic / immunology*
  • Recombinant Fusion Proteins / genetics*
  • Transduction, Genetic
  • Xenograft Model Antitumor Assays

Substances

  • Epitopes, T-Lymphocyte
  • Receptors, Immunologic
  • Recombinant Fusion Proteins
  • Receptor, ErbB-2