Figure 3

KPT-8602 efficiently targets the LICs of three primary AML samples engrafted into NSG mice. (a) Scheme for the limiting dilution repopulation assay. After 4 weeks of treatment, bone marrow AML cells isolated from mice treated with either vehicle, selinexor or KPT-8602 were re-transplanted at serial dilutions into new recipient animals. 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 drug-treated AML cell populations. (b) The table shows the LIC frequencies in AML-CN, AML-CK and AML/MDS xenografts after treatment with vehicle, selinexor or KPT-8602. The fold reduction in LIC frequency in response to drug treatment compared to vehicle controls shows the extent of LIC elimination in response to drug therapy.