Dr. Foued Ghanjati and Prof. Dr. Christian Flotho, of the Division of Pediatric Hematology and Oncology, have been awarded funding from the Deutsche Kinderkrebsstiftung for their project, “Clonal architecture in juvenile myelomonocytic leukemia”. This project addresses the role of intratumoral clonal diversity in disease progression and treatment resistance in JMML. The authors hypothesize that the early presence of certain subpopulations gives rise to therapy-resistant cell clones, which are a key factor in determining the risk of progression or relapse and hence the ultimate outcome. Previous characterization of the genetic landscape in JMML was based on bulk samples; however, the authors propose to generate genetic maps at the single-cell level from a well-documented patient cohort and associate these with hematologic and clinical information. The objective is to demonstrate differences in subclonal composition within NRAS-mutated JMML that does or does not progress under a watch-and-wait regimen. It is anticipated that the findings will assist in determining the risk of treatment failure at an early stage of JMML, thereby improving the clinical care of affected children and opening avenues to innovative therapeutic concepts.
