Therapeutic efficacy of NSC606985, a novel camptothecin analog, in a mouse model of acute promyelocytic leukemia

Nanomolar concentrations of NSC606985, a novel camptothecin (CPT) analog, effectively induces apoptosis in vitro in acute myeloid leukemic (AML) cells. Here we investigated the potential therapeutic effects of NSC606985 on the mice model with acute promyelocytic leukaemia (APL), a unique subtype of AML. The results showed that NSC606985 at single dose rapidly eliminated the infiltration with apoptosis induction of leukemic cells in peripheral blood and tissues. Treatment of NSC606985 with two regimens, continuous monotherapy and intermittent long-term therapy, significantly prolonged the survival of leukemic mice with tumor regression. These results propose that NSC606985 warrants further preclinical and clinical investigations for AML treatment.