In vivo therapeutic efficacy of MP-A08 in a human xenotransplantation model of AML. (A-C) NOD-SCID mice were engrafted with primary AML blasts, and after disease was established (2-6 weeks), mice were IP injected with either vehicle or MP-A08 (100 mg/kg) 6 times a week for 2 weeks. Engraftment was quantified by assessing the percentage of human CD45⁺ cells in the bone marrow of recipient mice. Each symbol represents the percentage of CD45⁺ cells observed in a separate mouse. Significance was assessed by Student t test. (D) Engrafted mice exhibited overt signs of AML including enlarged spleens. (E) Human cells in the bone marrow of engrafted mice were stained with the human specific mitochondrial antibody MTC02. (F) NOD-SCID mice were engrafted with primary AML blasts, and after disease was established, mice were injected IP 5 times/week for 32 days with 100 mg/kg MP-A08. P values were calculated using the log-rank Mantel-Cox test.