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Natural sciences
- Bio-informatics
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Medical and health sciences
- Medical genomics
- Oncology not elsewhere classified
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.