T-cell receptor gene therapy in human melanoma-bearing immune-deficient mice: human but not mouse T cells recapitulate outcome of clinical studies

Hum Gene Ther. 2012 Feb;23(2):187-201. doi: 10.1089/hum.2010.126. Epub 2011 Dec 2.

Abstract

Adoptive cell therapy using T-cell receptor (TCR)-engineered T cells is a clinically feasible and promising approach to target tumors, but is currently faced with compromised antitumor efficacies in patients. Here, we extensively validated immune-deficient mice to facilitate further development of the therapeutic potential of TCR-engineered T cells. Treatment of human melanoma-bearing SCID or NSG mice with high doses of human T cells transduced with an hgp100/HLA-A2-specific TCR did not result in antitumor responses irrespective of chemotherapeutic preconditioning. Imaging of human green fluorescent protein-labeled T cells demonstrated significant T-cell accumulation in intratumoral vasculature directly upon T-cell transfer, which was followed by loss of T cells within 72 hr. Peripheral persistence of human T cells was highly compromised and appeared related to T-cell differentiation. On the contrary, adoptive transfer (AT) of relatively low numbers of hgp100/HLA-A2 TCR-transduced mouse T cells resulted in rapid clearance of large established human melanomas. Unexpectedly and in contrast to reported studies with chimeric antibody receptor-engineered T cells, antitumor activity and homeostatic expansion of T cells were independent of TCR transgene as evidenced in two SCID strains and using two different human melanoma cell lines. Interestingly, the xeno-reactive melanoma response of mouse T cells appeared to be dictated by CD4(+) tumor-infiltrating lymphocytes and did not require in vitro T-cell activation, retroviral gene transfer, or subcutaneous interleukin-2 support. Taken together, AT of human but not mouse T cells in human melanoma-bearing immune-deficient mice is in close accordance with clinical studies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Differentiation
  • Genes, Reporter
  • Genetic Therapy*
  • Genetic Vectors
  • Green Fluorescent Proteins
  • HLA-A2 Antigen / genetics*
  • HLA-A2 Antigen / immunology
  • Humans
  • Immunotherapy, Adoptive*
  • Melanoma / immunology
  • Melanoma / pathology
  • Melanoma / therapy*
  • Mice
  • Mice, SCID
  • Receptors, Antigen, T-Cell / genetics*
  • Receptors, Antigen, T-Cell / immunology
  • Retroviridae / genetics
  • Species Specificity
  • T-Lymphocytes / immunology
  • T-Lymphocytes / metabolism
  • Transduction, Genetic
  • Transgenes
  • gp100 Melanoma Antigen / genetics*
  • gp100 Melanoma Antigen / immunology

Substances

  • HLA-A2 Antigen
  • Receptors, Antigen, T-Cell
  • gp100 Melanoma Antigen
  • Green Fluorescent Proteins