Extended Data Fig. 6: hCSF1R CAR T cells are effective in vivo.
(a) Treatment scheme used for PDX-372 model. (b–d) BLI images (b), BLI quantification of tumor-burden (c) and survival curves (d) of PDX-372 tumor-bearing mice injected with 2 × 106 hCSF1R, CD33 CART or control-transduced T cells (n = 5 mice per group). (b) White cross, censored mice; red cross, mice succumbed to disease. (e) IHC staining of human CSF1R in the bone marrow of control-treated PDX-372-bearing mice. Left: IHC for human CSF1R. Right: Isotype (top) and detection system control (bottom) for CSF1R IHC staining. (f) Schematic of treatment scheme used for OCI-AML3 cell line xenograft model. (g, h) BLI images (g) and survival curves (h) of OCI-AML3 tumor-bearing mice injected with 6 × 106 hCSF1R CART or control-transduced T cells (n = 3–4 mice per group). (g) Red cross, mice succumbed to disease. (a–h) Statistical significance was calculated using two-way ANOVA with Šidák multiple comparison correction. (i) log2 expression of CSF1R and CD86 target antigens or CD123 and CD33 controls in bulk RNA-sequencing dataset of the Leukemia MILE study (n = 615 different patients). HBM, healthy bone marrow. Data was obtained from bloodspot.eu. Dashed line represents the median, dotted line the interquartile ranges. Statistical significance was calculated using ordinary one-way ANOVA with Šidák multiple comparison correction. (j) Simple linear regression analysis of in vitro lysis of CAR T cells and target antigen density of the indicated AML cell line measured by flow cytometry. r = spearman correlation coefficient, p = p-value. Three independent antigen density measurements were used to perform regression analysis. For Kaplan-Meier-Curves, statistical significance was calculated with log-rank test.
