CD34−/lineage− cells from patient #25 were sorted into MUC1high and MUC1low populations. A. The CD34−/lineage− MUC1high cells were inoculated into 11 NSG mice (1×106/mouse). At 60 days after inoculation, the mice were treated with PBS or GO-203 administered subcutaneously daily for 21 days, and then sacrificed. Bone marrow cells from a representative PBS-treated mouse were analyzed for hCD45, hCD11C and hCD20 expression (A, left). The orange-P2 gate represents the homogenous hCD45+/hCD11C+ leukemia population (A, left). Bone marrow cells from a representative GO-203-treated mouse were analyzed for hCD45, hCD11C and hCD20 expression (A, right). B. The results are expressed as the percentage of hCD45+ leukemia cells in the bone marrows of the individual mice in the control and treated groups. The horizontal bar represents the mean percentage of hCD45+ cells. C. The CD34−/lineage− MUC1low cells were inoculated into 10 NSG mice (1×106/mouse). At 60 days after inoculation, the mice were treated with PBS or GO-203 administered subcutaneously daily for 21 days, and then sacrificed. Bone marrow cells from representative PBS- and GO-203-treated mice were analyzed for hCD45, hCD11C and hCD20 expression (C, left and right). D. The results are expressed as the percentage of hCD45+ leukemia cells in the bone marrows of the individual mice in the control and treated groups. The horizontal bar represents the mean percentage of hCD45+ cells. E–F. CD34−/lineage− cells from patient #26 that expressed high MUC1 levels in >90% of the population were inoculated into NSG mice (0.5×106/mouse). At 60 days after inoculation, the mice were treated with PBS or GO-203 administered subcutaneously daily for 21 days, and then sacrificed. Bone marrow cells from representative PBS- and GO-203-treated mice were analyzed for hCD45 expression (E, left and right). The results are expressed as the percentage of hCD45+ leukemia cells in the bone marrows of the individual mice in the control and treated groups (F). The horizontal bar represents the mean percentage of hCD45+ cells.