Ouabain (OUA) and pyrithione zinc (PZ) have been proved as the potential drugs for treating acute myeloid leukemia (AML). Selected from a screening among 1040 Food and Drug Administration-approved pharmacological agents, both drugs show ability to induce apoptosis of the culturing AML cells, exhibiting the poisoning effect on the cells. Studies also reveal the efficiency of the drugs in inhibiting the growth of human AML cells injected into the mice lacking of immunity and killing primary AML cells from the peripheral blood of AML patients1.

AML is a quickly progressive malignant disease characterized by too many immature blood-forming cells in the bone marrow and blood which interfere with the production of normal blood cells. In normal process of the formation and development of blood cells, a normal cell is gradually matured from immature blood-forming cells without uncontrolled growth. However, a single malignant AML cell accumulates genetic and/or epigenetic changes which “freeze” the cell in its immature state and prevent differentiation in AML2. When this is combined with other abnomal changes which disrupt genes controlling proliferation, the result is uncontrolled proliferation and/or abnormal survival of an immature clone of cells, leading to the clinical entity of AML3. The malignant cell often possesses surface marker CD34, a marker of primitive blood cells4. Therefore, to eliminate the malignant cells, a successful drug treatment of AML has to inhibit cell proliferation and/or induce cell death including apoptosis and necrosis. Since gene regulatory networks that govern these malignant cells are very complicated, the discovery of drugs that have different targets on AML cells is an attractive but difficult endeavor5.

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