While global survival of children with cancer is reaching up to 80%, cure rates remain disappointingly low for certain high risk types of tumours and for most relapsed cases. Moreover, studies on late effects of cancer treatment are showing high rates of chronic disease due to treatment related toxicities indicating the need for less toxic treatments in children. In a new kind of trials, called basket trials, cancer patients are treated according to a common genetic alteration as opposed to a pathological diagnosis, with the aim to explore the effectiveness of targeted therapy across several cancer types. Recent advances in genomic profiling techniques have enabled the study of cancer genomes with unprecedented detail and offer novel opportunities for precision medicine guided treatment. However, classical tumour biopsy sampling is invasive and consequently painful and even risky for the patient. Furthermore, serial sampling of tumour tissue and metastatic burden are not very well captured using tissue biopsies.
In my project, we aim to use liquid biopsies to monitor the drug response in paediatric patients included in basket trials. In this way, we hope to get insights into the mechanisms of drug efficacy to identify new biomarkers for disease and treatment response monitoring.