Immune responses to transgene and retroviral vector in patients treated with ex vivo-engineered T cells

Blood. 2011 Jan 6;117(1):72-82. doi: 10.1182/blood-2010-07-294520. Epub 2010 Oct 1.

Abstract

Adoptive transfer of immune effector cells that are gene modified by retroviral transduction to express tumor-specific receptors constitutes an attractive approach to treat cancer. In patients with metastatic renal cell carcinoma, we performed a study with autologous T cells genetically retargeted with a chimeric antibody receptor (CAR) directed toward carbonic anhydrase IX (CAIX), an antigen highly expressed in renal cell carcinoma. In the majority of patients, we observed distinct humoral and/or cellular anti-CAIX-CAR T-cell immune responses in combination with a limited peripheral persistence of transferred CAIX-CAR T cells in the majority of patients. Humoral immune responses were anti-idiotypic in nature and neutralized CAIX-CAR-mediated T-cell function. Cellular anti-CAIX-CAR immune responses were directed to the complementarity-determining and framework regions of the CAR variable domains. In addition, 2 patients developed immunity directed against presumed retroviral vector epitopes. Here, we document the novel feature that therapeutic cells, which were ex vivo engineered by means of transduction with a minimal γ-retroviral vector, do express immunogenic vector-encoded epitopes, which might compromise persistence of these cells. These observations may constitute a critical concern for clinical ex vivo γ-retroviral gene transduction in general and CAR-retargeted T-cell therapy in particular, and underscore the need to attenuate the immunogenicity of both transgene and vector.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adoptive Transfer
  • Amino Acid Sequence
  • Antigens, Neoplasm / genetics
  • Antigens, Neoplasm / immunology*
  • Carbonic Anhydrase IX
  • Carbonic Anhydrases / genetics
  • Carbonic Anhydrases / immunology*
  • Carcinoma, Renal Cell / immunology
  • Carcinoma, Renal Cell / therapy*
  • Cohort Studies
  • Genetic Engineering*
  • Genetic Vectors / immunology*
  • Humans
  • Kidney Neoplasms / immunology
  • Kidney Neoplasms / therapy*
  • Lymphocyte Activation
  • Molecular Sequence Data
  • Receptors, Antigen, T-Cell / immunology
  • Receptors, Antigen, T-Cell / metabolism
  • Retroviridae / genetics
  • T-Lymphocytes / immunology*
  • Transgenes / immunology*
  • Transgenes / physiology

Substances

  • Antigens, Neoplasm
  • Receptors, Antigen, T-Cell
  • CA9 protein, human
  • Carbonic Anhydrase IX
  • Carbonic Anhydrases