CART123 cells eliminate human AML in xenograft models. (A) Schematic of the MOLM14 xenograft model. NSG mice were sublethally irradiated (200 cGy) on day −1 and then injected via tail vein with 1 × 106 green fluorescent protein/luciferase+ MOLM14 on day 0. BLI was performed on day 6 to quantify engraftment and for randomization of treatment groups. Saline vehicle, CART19 cells (1 × 106), or CART123 (1 × 106) cells were injected IV on day 7, and mice were followed with serial BLI. Quantification of BLI radiance was used as a surrogate measurement of AML burden. (B) Elimination of MOLM14 occurred only in xenografted mice treated with CART123 cells, as measured by BLI radiance and displayed colorimetrically. (C) Summary BLI data from 3 MOLM14 xenograft experiments demonstrated rapid leukemic progression in vehicle-treated (black) and CART19-treated (blue) mice, whereas AML rejection was observed in CART123-treated mice (red). Mean radiance (symbols) with standard error of the mean (whiskers) are depicted at each time point. (D) Survival analysis of MOLM14 xenograft mice revealed significant survival for CART123-treated mice in comparison with vehicle- and CART19-treated mice. Attrition of CART123 T-cell–treated mice was primarily due to BLI-detectable AML progression in facial bones and subsequent anorexia and weight loss. Data were summarized from 4 independent experiments.
