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Drug platforms

Induced pluripotent stem (iPS) cell-derived, chimeric antigen receptor (CAR)-expressing T cells for immunotherapy

iPS cell-derived, CAR-expressing T cells could be used for immunotherapy in patients when suitable autologous or allogeneic T cells are unavailable. In peripheral blood T lymphocytes, viral vector-mediated expression of four factors reprogrammed the cells into iPS cells. These iPS cells were then transduced with a viral vector encoding a CD19-specific CAR and differentiated into highly cytotoxic T lymphocytes. In a mouse model of Burkitt's lymphoma, expanded, iPS cell-derived, CD19-specific CAR T lymphocytes conferred a survival advantage comparable to that of parent CD19-specific CAR T lymphocytes. Next steps include further optimization of the method for generating iPS cell-derived, CAR-expressing T cells for autologous or allogeneic therapies (see Supersizing adoptive T cell transfer, page 6).

SciBX 6(36); doi:10.1038/scibx.2013.1014
Published online Sept. 19, 2013

Patent application filed; available for licensing

Themeli, M. et al. Nat. Biotechnol.; published online Aug. 11, 2013;
Contact: Michel Sadelain, Memorial Sloan-Kettering Cancer Center, New York, N.Y.