Neuroblastoma is a malignant tumor of the peripheral nervous system and mainly affects toddlers and young children. In the EU region, there are 1500 new cases per year and around 50 percent of patients are high-risk cases. Recurrences occur frequently, and conventional therapies are no longer effective for these children.
With liquid biopsies, it is possible to monitor therapy success and predict tumor relapse in time to take medical countermeasures. Scientists from leading European research institutions in pediatric oncology are testing this promising diagnostic tool under the coordination of the European Society for Pediatric Oncology (SIOPE) and the scientific lead of the Princess Máxima Center for Pediatric Oncology and St. Anna Children's Cancer Research Institute (CCRI).
Why liquid biopsy?
Liquid biopsies are a promise for the future of personalized cancer medicine: Many children with high-risk neuroblastoma are at risk of not responding to therapy and developing a recurrence. Currently, the success of the therapy is monitored using medical imaging procedures and bone marrow examinations. These examinations are expensive, invasive, and stressful for children who need anesthesia.
Liquid biopsies use small blood samples, so the procedure is minimally invasive and comparatively simple. In the laboratory, the sample can be used to determine whether children have responded to therapies and detect an impending recurrence earlier.
Genetic markers in the blood plasma—cell-free DNA fragments and mRNA—that are released by the tumor cells and provide information about the genetic changes in the tumor are examined. In the event of a relapse, these can help to find targeted therapies for children with neuroblastoma. For example, the enzyme anaplastic lymphoma kinase (ALK) plays a decisive role in cancer development. If a child’s tumor has an aberration in the ALK gene, it could be targeted with ALK-inhibiting drugs.
Liquid biopsies in the clinical setting for the first time
A total of 25 leading European research institutions in pediatric oncology have now joined forces for an EU project funded by Horizon Europe. The aim is to study the benefit of these new diagnostic methods: Small blood samples will be collected several times from 150 patients with high-risk neuroblastoma and will be examined in laboratories, such as the Princess Máxima Center and Labdia Labordiagnostik GmbH.
The goal of the project MONALISA, or monitor neuroblastoma relapse with liquid biopsy sensitive analysis, is to develop a clinical study that can be quickly implemented in clinical practice. "And this is already the first special feature," says Sabine Taschner-Mandl, PhD, principal investigator at the CCRI, who is co-leading liquid biopsy diagnostics in MONALISA. “We are using liquid biopsies for children with neuroblastoma for the first time in a clinical setting. So far, we have only tested them in research [settings]," says the tumor biologist.
Lieve Tytgat, MD, PhD, a pediatric oncologist specializing in neuroblastoma at the Princess Máxima Center and scientific co-lead of the MONALISA project, says, “This research is a real breakthrough in the world of noninvasive diagnostics. Liquid biopsies are increasingly commonly used in adults with cancer. With this study, we aim to catch up with research in adults, an important development for children with cancer.”
MONALISA as a standard procedure
Monitoring the response to therapy and disease course is pivotal for improving the chance in survival of patients with high-risk neuroblastoma. MONALISA aims to close existing gaps in diagnostics and establish liquid biopsies as a standard procedure for monitoring relapsed neuroblastoma.
The method could serve as a blueprint for other pediatric cancers. MONALISA also aims to analyze how patients and parents experience the use of liquid biopsies to be able to adapt the procedure in the future. With the project, the scientists are getting one step closer to effective personalized medicine in children with neuroblastoma and will offer hope for a better chance to be cured.
- This press release was originally published on the Princess Máxima Center for Pediatric Oncology website