Advancing liquid biopsies for monitoring and personalized treatment of children with neuroblastomas
The embryonal tumor, neuroblastoma, accounts for 11% of all cancer-related deaths in children. Its heterogeneous tumor biology creates clinical variability spanning spontaneous regression to rapid metastasizing progression. Long-term survival of high-risk disease remains poor, with <40% overall survival after first-line treatment and <10% after relapse, despite considerable international efforts to improve treatment over the last decades. Liquid biopsies have the power to revolutionize clinical care for children with high-risk neuroblastoma by reflecting precise disease status at any time during treatment and care. Blood and bone marrow samples are a less invasive source of biomarkers for patient monitoring and therapeutic decision-making. The LIQUIDHOPE consortium combines internationally recognized experts in neuroblastoma pan-omics and computational discovery with leading pediatric oncologists to advance this emerging clinical paradigm change. LIQUIDHOPE aims to accelerate transfer of liquid biopsy approaches into the clinic within 3 parallel research arms designed to overcome current hurdles in (1) therapy response assessment, (2) minimal residual disease (MRD) monitoring and (3) actionable target identification, and define the best marker/analysis method or combination thereof for patient monitoring as its secondary aim. LIQUIDHOPE will apply targeted metabolomics; cfDNA whole-exome sequencing; cfDNA transcriptional start site and methylation profiling; unbiased total RNA profiling to monitor long noncoding and circular RNA disease markers; droplet digital PCR of DNA/RNA disease markers; automated multiple marker imaging and sophisticated bioinformatics. LIQUIDHOPE can identify and validate predictive markers for treatment response, MRD, relapse and treatment choice in blood/bone marrow surrogates to advance unique liquid biopsy-based innovations for patient monitoring and personalized treatment of children battling neuroblastoma.
Funded by the European Union under the Horizon Europe Framework Programme - Grant Agreement Nº: 101095654. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or European Health and Digital Executive Agency (HADEA). Neither the European Union nor the granting authority can be held responsible for them.