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A SIOPEN pragmatic clinical trial to MOnitor NeuroblastomA relapse with LIquid biopsy Sensitive Analysis

Project description

Liquid biopsies for monitoring relapsed neuroblastoma in children

Neuroblastoma accounts for 15 % of cancer-related deaths in children. Current standard-of-care monitoring methods are invasive and burdensome to children and their families. The EU-funded MONALISA project aims to introduce minimally invasive liquid biopsies to monitor relapsed or refractory neuroblastoma, serving as a model for other paediatric cancers. The primary goal is to investigate whether liquid biopsies may detect relapse or disease progression earlier than the standard-of-care methods. The project will provide reliable early assessments of molecular progression through a pragmatic clinical trial. Importantly, it will develop a digital decision-support tool to help clinicians interpret liquid biopsy results and a remote patient reporting app to assess the effect of frequent liquid biopsy tests in the quality of life of patients and their families.

Objective

High-risk neuroblastoma accounts for 15% of cancer related-deaths in children. Half of the >1500 patients yearly diagnosed with neuroblastoma in the EU have high-risk disease, which will relapse or progress in half these cases after first-line treatment. Relapsed neuroblastoma is aggressive and often therapy-resistant. Monitoring for disease relapse and therapy response is crucial for the survival chance of these patients. The current standard-of-care for monitoring are imaging technologies and bone marrow assessment, which are costly, invasive and a burden for children, who often require anesthesia. These drawbacks limit how often is monitored. More sensitive, less invasive and less toxic monitoring techniques are needed. The mutational spectrum often changes in recurring tumors, which may explain therapy resistance and provide additional druggable targets. Imaging, however, provides no information about molecular characteristics. Liquid biopsy tests are minimally invasive, allow frequent sampling and sensitively detect tumor molecular markers in tumor-derived DNA and messenger RNA circulating in peripheral blood. MONALISA aims to close existing gaps and establish liquid biopsies as standard-of-care to monitor relapsed/refractory neuroblastoma, as a blueprint for other pediatric cancers. Reliable, early assessment of molecular progression or relapse is the main aim of the pragmatic randomized clinical trial proposed in MONALISA. We develop a digital decision support tool to help oncologists use the new monitoring and apply patient-reported outcomes to integrate patient viewpoints and assess the effect of minimally invasive, liquid biopsy diagnostics on quality of life. We will establish whether events can be detected earlier using liquid biopsy monitoring, and whether better overall survival is enabled by earlier diagnosis and treatment interventions. This essential step towards personalized medicine will support reliable disease monitoring under treatment. “This action is part of the Cancer Mission cluster of projects on ‘‘Diagnostics and Treatment (diagnostics).”

Fields of science (EuroSciVoc)

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Programme(s)

Coordinator

SIOP EUROPE
Net EU contribution
€ 277 283,50
Address
CLOS CHAPELLE-AUX-CHAMPS 30
1200 Bruxelles / Brussel
Belgium

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Region
Région de Bruxelles-Capitale/Brussels Hoofdstedelijk Gewest Région de Bruxelles-Capitale/ Brussels Hoofdstedelijk Gewest Arr. de Bruxelles-Capitale/Arr. Brussel-Hoofdstad
Activity type
Other
Links
Total cost
€ 277 283,50

Participants (20)

Partners (4)

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