Fig. 1

In vitro and in vivo efficacy profile of TEG001. (a) Antitumor reactivity of TEG001 towards patient-derived primary AML blasts in vitro. Effector cells TEG001 and primary AML blasts from multiple donors (E:T ratio is 1:3) were incubated for 24 h with or without 10 μM PAM as indicated. Daudi and healthy T cells were included as positive and negative target controls, respectively. IFNγ secretion was measured by ELISPOT. IFNγ spots per 15,000 T cells are shown as mean ± SD of at least 3 independent replicates for each target. Fifty spots/15,000 cells were considered as a positive antitumor response and indicated by the black horizontal line. Statistical significances were calculated by two-way ANOVA; *, P < 0.05; **, P < 0.01; ***, P < 0.001; ****, P < 0.0001; (b) Bulk αβT cells (as mock T cells) or TEG001 cells were incubated with primary AML blast from donor p2 for 5 h at E:T ratio 10:1. Then cells were plated in methylcellulose-based medium and, after 8 days, colony formation was quantified using an inverted microscope. Shown is the number of CFU formed. Data is the result of a single experiment from single primary AML donor; (c) Schematic overview of in vivo experiment. NSG mice were irradiated at day 0 and engrafted with primary AML cells at day 1. AML cells were followed-up in the peripheral blood by flow cytometry. When the average AML cells were > 500 cells/ml, treatment was initiated. Mice received 2 injections of therapeutic TEG001 or TEG-LM1 mock in the presence of PAM (at week 7 and 9) and IL-2 (at week 7); (d) Tumor burden for primary AML was measured in peripheral blood by quantifying for absolute cell number by flow cytometry. Data represent mean ± SD of all mice per group (n = 5). Statistical significances were calculated by non-parametric 2-tailed Mann-Whitney t-test; *, P < 0.05; **, P < 0.01; ***, P < 0.001; ****, P < 0.0001