Combinatorial inhibition of AMPK and BET prolongs survival in patient-derived xenograft AML model. (A-B) MOLM13 cells were treated with JQ1 (0.05 ng/μL) and an AMPK inhibitor compound C (0.1 ng/μL) individually or in combination and cell proliferation (A) and expression of a myeloid differentiation marker CD33 (B) were analyzed (n = 3). (C-D) Survival curve of NSG-SGM3 mice treated with JQ1 (2.5 mg/kg/d) and compound C (1 mg/kg/d) individually or in combination after transplantation of MOLM13 cells (C) (n = 8) or patient-derived t(9;11) AML cells (D) (n = 8). (E) Flow cytometry analysis of mice described in (D) to detect human CD45⁺ cells in the peripheral blood (n = 3). (F) Quantitative PCR using human AML cells isolated from recipient mice as shown in (D) to detect the expression of key leukemogenic genes (n = 3). All data represent mean ± standard deviation. *P < .05; **P < .01; ***P < .001 by 1-way ANOVA, except for comparison of the survival curves in which the significance was accessed by a log-rank test.