In children, acute myeloid leukemia (AML) is a form of blood cancer still associated with very low cure rates. Although less frequent than other pediatric leukemia subtypes, AML is responsible for the majority of deaths in children. AML is a disease composed of various genetic subgroups, some of which are associated with a very poor prognosis, with survival rates of around 20%. Clinically, however, all children receive the same combination of drugs, regardless of AML genetic subtype, given the absence of targeted therapy. This means that children are not cured, just as they do not receive treatment tailored to their disease. The fact that the various AML subtypes are relatively rare represents a major challenge for studying the disease and identifying new therapeutic targets.

The aim of this project is to identify new drugs to improve survival rates in high-fatality childhood AML. To achieve this, the team will combine two innovative strategies. Firstly, they will use unique models of pediatric AML that they have derived from cord blood cells, thus compensating for the small number of patient samples available. The study will be complemented by a provincial biobank (Sainte-Justine, MUHC) set up to carefully collect cases of pediatric AML. Secondly, they will use modern technological platforms in place at IRIC (Institute for Research in Immunology and Cancer) to test the anti-leukemic potential of thousands of chemical compounds. Using a large-scale screening technology approach, they will also ablate each gene individually in leukemia cells to identify the genetic determinants of AML. As a multidisciplinary team, their common goal is to identify new targeted therapies in pediatric AML that can be rapidly transferred to the clinic to improve survival rates in a disease that is still too lethal.