Modulating microRNA-193a expression levels as a treatment for acute myeloid leukemia (AML)

Acute myeloid leukemia (AML) has a dismal prognosis in Canada with only every 5th patient surviving 5 years. To find novel treatment options, we explore the therapeutic potential of the tumor suppressor microRNA (miR)-193a in AML patients together with InteRNA, a company that developed a novel drug based on the liposomal encapsulation of miR-193a (1B3), which showed very promising preclinical results in solid tumors and provided the rational for a phase I trial starting in spring 2020. We and others have previously shown that miRNAs are small RNAs that impact leukemia cells and are an emerging class of drugs. Recent data from our group showed a strong leukemia inhibition via miR-193a in animal AML models, highlighting the tumor suppressive effect of this miRNA. In addition, we are studying the regulation of miR-193a in AML cells to develop strategies to reinstate miR-193a expression and thus enhance its tumor suppressor function. This innovative study pioneers a novel class of RNA-based drugs in the treatment of AML and the groundwork for future clinical trials.