Ide significantly enhances the anti-leukemic function of DNTs in PDX model. (A) Schematic diagram of patient-derived xenograft (PDX) model used. Sublethally irradiated NSG mice were infused with 2–3 × 106 primary AML patient sample on day 0. On day 17 and 20, mice were infused with 2 × 107 ctrl-DNTs or Ide-DNTs. Mice were sacrificed and AML engraftment were assessed in various tissues on day 24 or day 34. (B) NSG mice engrafted with a primary AML sample (090543) were treated with PBS, ctrl-DNT, or Ide-DNT as described in Figure 2A. AML engraftment and counts in bone marrow were assessed on day 34. Representative flow plots show AML engraftment (CD45+ CD33+) in the BM. Dot plot shows the frequency and number of AML cells engrafted in the BM. Horizontal line represents the mean of BM AML engraftment level, each symbol represents individual mice, and error bars represent SEM. The results shown are representative of two independent experiments. (C) Percent reduction in AML engraftment from two PDX experiments performed using different primary AML patient samples (090543 and 130624). Horizontal bar represents the mean of reduction in AML engraftment level in BM on day 34 relative to the untreated group in ctrl-DNT (blue)- and Ide-DNT (red)-treated groups. Each symbol represents individual mice and error bars represent SEM. (D) Sublethally irradiated (250 cGy) NSG mice were engrafted with one of four primary AML samples (sample ID: 150935, 130578, 090543, and 130624; 2–3 × 106 cells/mouse). Subsequently, mice were treated with PBS (black) or 1.5–2 × 107 cells per infusion of ctrl-DNTs (blue) or Ide-DNTs (red) on day 17 and 20. For the PDX model using the sample IDs 150935 and 130578, BM AML engraftment was assessed on day 24, which was 4 days post-treatment. For the sample IDs 090543 and 130624, BM AML engraftment was assessed on day 34, which was 14 days post-treatment. Each group had 4–8 mice. Horizontal bar represents the mean of BM AML engraftment level normalized to the untreated group, and error bars represent SEM. * p < 0.05, ** p < 0.01, *** p < 0.001.