Acute myeloid leukemia (AML) is a type of blood cell cancer characterized by clonal expansion of functionally immature myeloid precursors accumulating within the bone marrow, peripheral blood, and infrequently other tissues, resulting in impaired hematopoiesis. In Canada, the 5-year survival for AML patients is 21%, requesting new treatment approaches. MicroRNAs (miRNAs) are small non-coding RNAs that impact post-translational processing in normal and malignant processes. Their ability to modulate individual pathways at various levels or many pathways simultaneously gives them a central role in developmental, physiological, and pathological processes.
We explore miR-193a as a potential therapeutic in AML. Our preliminary data showed a significant repression of miR-193a in AML patients. The engineered over-expression of miR-193a strongly impaired leukemogenesis in animal models. In addition, we are studying the regulation of miR-193a via WT1 in AML cells to develop strategies to reinstate miR-193a expression and thus its tumor suppressor function. This study pioneers a novel class of RNA-based drugs in the treatment of AML and the groundwork for future clinical trials.