BT4ChildLC is a research program aimed at identifying predictive biomarkers and new therapies to fight childhood and adolescent liver cancer more effectively.
About Pediatric Liver Cancer
In children, malignant tumors originating in the liver are rare, accounting for 1% of pediatric cancers. In 90% of cases, these are either hepatoblastomas (HB, 2/3) or hepatocarcinomas (HCC, 1/3). This cancer is suspected in children if there is an increase in abdominal volume and pain or digestive disorders. The diagnosis is confirmed by radiological examinations (ultrasound, CT scan, or MRI), and the treatment is primarily surgery combined with chemotherapy, both before and after the operation.
The prognosis for hepatoblastomas is better than for hepatocarcinomas, so it is important to better understand these rare pathologies to improve treatment outcomes. Indeed, more than 40% of patients develop severe late effects from the treatment such as deafness, heart failure, and secondary tumors. Conversely, the majority of HCC patients (generally resistant to chemotherapy) have poor survival unless the tumor is completely removed, which occurs in less than 20% of cases.
Overall, about one in five children with liver tumors presents an aggressive form and does not survive the disease because the cancer does not respond to current treatments or relapses. Although clinical characteristics conferring good and poor risk are known, no biological factors are used in managing these patients to decide the intensity of the treatment they receive. The main reason preventing the improvement of pediatric liver cancer treatment is the lack of robust biological and pathological studies using a large number of diagnostic biological samples from these patients.
About BT4ChildLC
Due to the low incidence of pediatric liver cancer, progress in its treatment requires international collaborative efforts. A clear example of success is PHITT (Paediatric Hepatic International Tumour Trial), the largest clinical study conducted in children worldwide, launched in Europe in 2017. Through PHITT, researchers are systematically collecting, for the first time in Europe, diagnostic samples of tumor tissues and blood from a large number of patients diagnosed with liver cancers in over 150 hospitals across 12 countries (as of December 2021, samples have been collected from over 200 patients).
In parallel, ambitious biological and pathological studies have been developed in conjunction with the PHITT trial thanks to funding obtained from the European Commission (H2020 ChiLTERN project, 2016-2021). This project aims to identify new markers of poor response to treatments and new therapies for these patients.
Thus, the current project leverages the largest European collection of pediatric liver cancer patient samples enrolled in the first international clinical trial, PHITT. The objectives of this project are to:
- Define biological factors (new stratification system) that can help clinicians offer the most appropriate treatment to each patient and, consequently, reduce harmful side effects and increase their chances of survival.
- Improve the understanding of the biology of highly aggressive tumors and identify key targets for new therapies using advanced molecular studies and machine learning tools.
- Identify new drugs for aggressive tumors by treating innovative models derived from pediatric liver cancer patients that we have already established.
Project Progress
The project has started well, and biomarker analyses have been conducted on nearly 100 children. Genetic and transcriptomic analyses are ongoing.
Summary of the BT4ChildLC Project
- Lead Investigators: Prof. Carolina Armengol
- Program Duration: January 2023 – January 2025
- Countries Involved: Spain, Italy, UK, Germany, Netherlands
- Funding by Fight Kids Cancer: €500,000
