New Investigator Research Grant Program

Acute leukemia is the most common form of childhood cancer, with acute myeloid leukemia (AML) being the leading cause of leukemia-related death in children. The genetic mutations that mistakenly attach two genes together can lead to the production of cancer-promoting fusion proteins and thus AML development. AML driven by the CBFA2T3-GLIS2 (CG) fusion is associated with a poor prognosis, with only 30% of affected children being cured by current standard therapies. Therefore, novel therapeutic approaches are needed to tackle this challenging disease. Our preliminary studies have uncovered that a protein responsible for transporting a nutrient called choline into cancer cells can be a potential target. Inhibiting this protein and thus blocking the choline supply can effectively repress the growth of CG-mediated AML. In this project, we will investigate and understand why choline is essential for CG-driven AML and why this AML type heavily depends on that particular transporter protein for choline supply. Additionally, we will assess whether targeting the choline transporter protein can be a promising therapeutic strategy in advanced leukemia experimental models. This research will pave the way for developing novel treatments against this aggressive pediatric cancer.