3D-organoid culture supports differentiation of human CAR+ iPSCs into highly functional CAR T cells

Cell Stem Cell. 2022 Apr 7;29(4):515-527.e8. doi: 10.1016/j.stem.2022.02.009. Epub 2022 Mar 11.

Abstract

Unlimited generation of chimeric antigen receptor (CAR) T cells from human-induced pluripotent stem cells (iPSCs) is an attractive approach for "off-the-shelf" CAR T cell immunotherapy. Approaches to efficiently differentiate iPSCs into canonical αβ T cell lineages, while maintaining CAR expression and functionality, however, have been challenging. We report that iPSCs reprogramed from CD62L+ naive and memory T cells followed by CD19-CAR engineering and 3D-organoid system differentiation confers products with conventional CD8αβ-positive CAR T cell characteristics. Expanded iPSC CD19-CAR T cells showed comparable antigen-specific activation, degranulation, cytotoxicity, and cytokine secretion compared with conventional CD19-CAR T cells and maintained homogeneous expression of the TCR derived from the initial clone. iPSC CD19-CAR T cells also mediated potent antitumor activity in vivo, prolonging survival of mice with CD19+ human tumor xenografts. Our study establishes feasible methodologies to generate highly functional CAR T cells from iPSCs to support the development of "off-the-shelf" manufacturing strategies.

Keywords: 3D-organoid culture; CAR; PSC-ATO; chimeric antigen receptor T cells; human iPSC; immunotherapy; off-the-shelf; pluripotent stem cell-artificial thymic organoid culture.

Publication types

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

MeSH terms

  • Animals
  • Cell Differentiation
  • Humans
  • Immunotherapy
  • Immunotherapy, Adoptive / methods
  • Induced Pluripotent Stem Cells* / metabolism
  • Mice
  • Organoids / metabolism
  • Receptors, Chimeric Antigen* / metabolism

Substances

  • Receptors, Chimeric Antigen