Manufacture of clinical-grade CD19-specific T cells stably expressing chimeric antigen receptor using Sleeping Beauty system and artificial antigen presenting cells

PLoS One. 2013 May 31;8(5):e64138. doi: 10.1371/journal.pone.0064138. Print 2013.

Abstract

Adoptive transfer of T cells expressing a CD19-specific chimeric antigen receptor (CAR) is being evaluated in multiple clinical trials. Our current approach to adoptive immunotherapy is based on a second generation CAR (designated CD19RCD28) that signals through a CD28 and CD3-ζ endodomain. T cells are electroporated with DNA plasmids from the Sleeping Beauty (SB) transposon/transposase system to express this CAR. Stable integrants of genetically modified T cells can then be retrieved when co-cultured with designer artificial antigen presenting cells (aAPC) in the presence of interleukin (IL)-2 and 21. Here, we reveal how the platform technologies of SB-mediated transposition and CAR-dependent propagation on aAPC were adapted for human application. Indeed, we have initiated clinical trials in patients with high-risk B-lineage malignancies undergoing autologous and allogeneic hematopoietic stem-cell transplantation (HSCT). We describe the process to manufacture clinical grade CD19-specific T cells derived from healthy donors. Three validation runs were completed in compliance with current good manufacturing practice for Phase I/II trials demonstrating that by 28 days of co-culture on γ-irradiated aAPC ∼10(10) T cells were produced of which >95% expressed CAR. These genetically modified and propagated T cells met all quality control testing and release criteria in support of infusion.

Publication types

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

MeSH terms

  • Antigen-Presenting Cells / cytology
  • Antigen-Presenting Cells / immunology
  • Antigen-Presenting Cells / metabolism*
  • Antigens, CD19 / genetics*
  • Antigens, CD19 / immunology
  • CD28 Antigens / genetics
  • CD28 Antigens / immunology
  • CD3 Complex / genetics
  • CD3 Complex / immunology
  • Cell Proliferation
  • Clinical Trials as Topic
  • Coculture Techniques
  • DNA Transposable Elements / genetics*
  • DNA Transposable Elements / immunology
  • Electroporation
  • Gene Expression
  • Gene Transfer Techniques
  • Genetic Therapy / methods
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunotherapy, Adoptive / methods
  • Interleukin-2 / genetics
  • Interleukin-2 / immunology
  • K562 Cells
  • Leukemia, B-Cell / immunology
  • Leukemia, B-Cell / pathology
  • Leukemia, B-Cell / therapy*
  • Lymphocyte Activation
  • Mutant Chimeric Proteins / genetics
  • Mutant Chimeric Proteins / immunology
  • T-Lymphocytes / cytology*
  • T-Lymphocytes / immunology
  • T-Lymphocytes / metabolism*
  • T-Lymphocytes / transplantation
  • Transposases / genetics
  • Transposases / immunology

Substances

  • Antigens, CD19
  • CD28 Antigens
  • CD3 Complex
  • CD3 antigen, zeta chain
  • DNA Transposable Elements
  • Interleukin-2
  • Mutant Chimeric Proteins
  • Transposases