In children, acute myeloid leukemia (AML) is a form of blood cancer that is still associated with low cure rates. Although not as frequent as other subtypes of pediatric leukemia, it accounts for most of the deaths related to the disease in children. AML is a disease composed of distinct genetic subtypes, and for some of them, survival rates can be dismal, and as low as 20%. Yet, all children in the clinic are treated with the same combination of drugs, irrespective of their disease subtype, because specific drugs are not currently available. Therefore, children are not cured because they are not receiving the combination of drugs that is appropriate for their disease subtype. The challenge in identifying promising leukemia specific drugs for pediatric AML resides in the fact that all the disease subtypes are relatively rare and thus difficult to study.
The team of Prof. Wilhelm, Dr. Barabé and Dr. Cellot aims to identify new drugs to improve survival in high fatality pediatric AML. To do so, they will combine two innovative strategies. First, their team will use unique AML models they have engineered from cord blood cells to overcome the paucity of patient samples. Studies will be complemented with primary patient samples that are collected through a provincial (Sainte-Justine, MUHC) biobanking initiative. Second, they will use modern facilities that are in place at the IRIC (Institut de Recherche en Immunologie et Cancer) to test thousands of drugs for activity against AML. Using a similar large scale screening technology, they can also delete individual genes in leukemic cells to identify specific genetic determinants of the disease. Overall, their goal as a multidisciplinary team is to identify novel promising drugs that are readily amenable to the clinic to improve cure rates in pediatric AML.