Targeting Leukemia Stem Cells in T-ALL with Amiloride

Despite advancements in treatment, T-cell acute lymphoblastic leukemia (T-ALL) remains difficult to cure when it relapses, largely due to leukemia stem cells (LSCs), a small but powerful group of cells that resist therapy and restart the disease. Using a zebrafish T-ALL model, we screened over 700 FDA-approved drugs and discovered that Amiloride can block LSC self-renewal by disrupting mitochondrial function. We received funding from the COBRE Center for Cancer and Metabolism for a pilot project to explore how Amiloride affects leukemia metabolism and advance preclinical testing in mouse and patient-derived models. Our goal is to bring this promising treatment strategy one step closer to the clinic, improving outcomes for children battling relapsed T-ALL.