Translational research in pediatric oncology (RTOP)

The main objectives of the RTOP (“Recherche Translationelle en Oncologie Pédiatrique”) research team is to promote translational research in two aggressive pediatric cancers, neuroblastoma (NB) and rhabdoid tumours (RT). Although different genetic alterations drive oncogenesis in NB and MRT, in both pathologies molecular
characterization and understanding of the underlying mechanisms leading to the observed phenotype is crucial for the development of new therapeutic approaches. Since its establishment in 2018, the research developed by the RTOP team has progressively evolved into two distinct research programs.

In the group led by Gudrun Schleiermacher, we address questions of genetic and epigenetic heterogeneity, clonal evolution, and high through-put drug screening including combinations and sequential therapies, with an aim to develop new therapeutic strategies in neuroblastoma and other highly aggressive pediatric cancers.
Furthermore, a focus of the team has been the development of liquid biopsies (ctDNA) as a means of studying clonal evolution and mechanisms of resistance. 

In the group led by Franck Bourdeaut focusing on rhabdoid tumors and SMARCB1-deficient cancers, the main goals were i) to understand the origin of these enigmatic cancers of early-onset and generate the most relevant preclinical genetically engineered mouse models to address this issue, ii) to take advantage of these
immunocompetent mouse models and the broad collection of fresh frozen or freshly resected human samples to describe the immune response acting in these tumors and to design immune-based preclinical therapies, and iii) to explore epigenetic vulnerabilities in the context of very simple genomes that characterizes these tumors.

The RTOP team identity is shaped by its tight links with the clinical pediatric department (SIREDO Integrated Pediatric Oncology center)  to which the two PIs belong. The scientific programme is thus also orientated towards clinical applications and the attractiveness of the lab is in addition defined by its interface position between experimental research and clinical applications