Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells

Proc Natl Acad Sci U S A. 2011 Dec 20;108(51):E1408-16. doi: 10.1073/pnas.1115050108. Epub 2011 Nov 28.

Abstract

The goal of cancer immunotherapy is the generation of an effective, stable, and self-renewing antitumor T-cell population. One such approach involves the use of high-affinity cancer-specific T-cell receptors in gene-therapy protocols. Here, we present the generation of functional tumor-specific human T cells in vivo from genetically modified human hematopoietic stem cells (hHSC) using a human/mouse chimera model. Transduced hHSC expressing an HLA-A*0201-restricted melanoma-specific T-cell receptor were introduced into humanized mice, resulting in the generation of a sizeable melanoma-specific naïve CD8(+) T-cell population. Following tumor challenge, these transgenic CD8(+) T cells, in the absence of additional manipulation, limited and cleared human melanoma tumors in vivo. Furthermore, the genetically enhanced T cells underwent proper thymic selection, because we did not observe any responses against non-HLA-matched tumors, and no killing of any kind occurred in the absence of a human thymus. Finally, the transduced hHSC established long-term bone marrow engraftment. These studies present a potential therapeutic approach and an important tool to understand better and to optimize the human immune response to melanoma and, potentially, to other types of cancer.

Publication types

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

MeSH terms

  • Animals
  • Antigens, CD34 / biosynthesis
  • Antineoplastic Agents / pharmacology*
  • CD8-Positive T-Lymphocytes / cytology*
  • CD8-Positive T-Lymphocytes / immunology
  • Cell Line, Tumor
  • Flow Cytometry / methods
  • Genetic Engineering / methods
  • Hematopoietic Stem Cells / cytology*
  • Humans
  • Immunotherapy / methods
  • Lymphocytes, Tumor-Infiltrating / cytology
  • Mice
  • Mice, SCID
  • Models, Genetic
  • Neoplasm Transplantation
  • Stem Cells / cytology
  • Thymus Gland / metabolism
  • Transgenes

Substances

  • Antigens, CD34
  • Antineoplastic Agents