Acute myeloid leukemia (AML)

Pediatric acute myeloid leukemia (AML) is a rare hematological disease that accounts for 20% of all pediatric leukemias. With the current treatment protocols, the five-year overall survival rates have reached a plateau of approximately 75%. In addition, 25% to 30% of the good responders will experience relapse, often correlated with specific genetic subgroups. 

Over the past years we have been actively identifying novel therapeutic targets for the eradication of leukemic stem cells (LSC) in acute myeloid leukemia (AML). We have developed treatment options including specific cytotoxic T-cells showing promising results in vitro which are currently validated in vivo. Specifically, following the identification of TARP, a novel therapeutic target in AML, we have been able to generate TARP-directed therapy (cytotoxic T-cells and nanobodies) and illustrated the therapeutic targetability (oral presentation during international conference Childhood Leukemia Lymphoma Symposium, May 2023). We recently added the focus on the epigenome and to non-coding genes expression. Therefore, we have also explored the non-coding transcriptome of AML. This resulted in an unique set of LSC- and leukemic myeloblast-specific long non-coding RNA (lncRNA) who are currently further investigated (Vanhooren et al., 2022). Additionally, we recently established a novel lncRNA expression signature, that allows to predict relapse-free survival in pedAML. Finally, during the past years we have established a repository of AML patient-derived xenografts at Ghent University Hospital. This will allow us and collaborating research teams, to perform extensive in vivo evaluations of novel therapeutics.


Vanhooren J*, Dobbelaere R*, Derpoorter C*, Deneweth L*, Van Camp L, Uyttebroeck A, De Moerloose B and Lammens T. (2023). CAR-T in the treatment of acute myeloid leukemia: barriers and how to overcome them. Hemasphere 7 (9): e937. DOI: 10.1097/HS9.0000000000000937. (* These authors contributed equally). PMID: 37674860