Figure 5.

Selinexor (KPT-330) targets the LICs of three primary AML samples engrafted into NSG mice. (a) Scheme for the limiting dilution transplantation assay. Bone marrow AML cells in xenografts treated with either vehicle or selinexor were re-transplanted at different serial dilutions into new recipient mice. The number of leukemic mice per total number of animals injected with AML cells in secondary recipients was used to determine the LIC frequency in vehicle- and selinexor-treated AML cell populations. LIC frequency of AML cells in AML-CK1 (b), AML-CK2 (c), and AML-CN (d) xenografts after treatment with either vehicle or selinexor. The table shows the number of leukemic mice in each secondary recipient group and reports the LIC frequencies with 95% confidence intervals for the three xenografts. *Pilot experiment for xenograft AML-CN (d), which demonstrated that 10³ hCD45+ cells isolated from vehicle-treated mice were sufficient to initiate leukemia in secondary recipients, was used to guide the hCD45+ cell doses for determining the LIC frequencies in this xenograft.