Pluripotin treatment suppresses refractory/relapsed primary human leukemia. (A) Experimental design for the analysis of pluripotin treatment on AML cells from the refractory/relapsed patients. Shown is the survival of NSGS mice transplanted with primary human AML cells from AML1 (B) and AML4 (C). Patient AML1 has only FLT3^ITD mutation though AML4 harbors mutation in FLT3^ITD, RAS^G13D, and PTPN11^D61V (supplemental Figure 5A). Note, pluripotin treatment with pluripotin significantly prolonged the survival, whereas gilteritinib was ineffective. Shown are leukemic burden measured by hCD45 levels in mice recipients of AML1 (D) and AML4 (E). Presented data are from 2 independent experiments (3 mice per group) shown as mean ± SD. ∗P < .05, ∗∗P < .01, and ∗∗∗P < .001. ns, not significant.