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Therapeutic Programs: Acute Myeloid Leukemia

Non-Small Cell Lung Cancer  |  Metastic Prostate Cancer  |  Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a genetically and phenotypically heterogeneous disorder of the hematopoietic stem cells.  It is characterized by failure of blood cells to differentiate, which results in the accumulation of non-functional cells called myeloblasts. AML includes multiple sub-groups that are classified cytogenetically. These include: (1) AML with normal karyotype, which accounts for 40-45% of the cases; (2) AML with primary balanced translocations, which is found in about 20-25% of the cases; and (3) AML with complex aberrant karyotypes, which is present in about 35-40% of all the AML cases.  The currently available treatments halt disease in only about one-third of the AML patients within the 18-60 age group. Those who respond to therapy never attain disease-free status. The percentage of relapse deaths observed in complete remission cases is very high.  Recent innovations in therapeutic design have led to the development of several AML drugs that are currently in clinical trails.  The heterogeneity of AML and diverse patient response and poor survival rate in adults adds impetus to the discovery of new molecular therapeutic targets.

We have used array and qRT-PCR analyses to evaluate the expression of miRNAs in the peripheral blood of more than 100 patients with acute myeloid leukemia.  Functional studies in five different leukemia cell lines have revealed that miRNA mimetics for let-7 and a second miRNA reduce the viability and proliferation of leukemic cancer cells.  Interestingly, these miRNAs do not have significant effects on normal T-cells, suggesting that the effect observed in the leukemia cells is cancer-specific.  Xenograft studies indicate that mimetics for let-7 and a second miRNA affect the viability of leukemia cells in vivo.  Our data suggest that one or two miRNA mimetics have the potential to specifically reduce the viability of leukemic cells and might be used to treat AML patients.