European scientists target high-risk childhood cancer with liquid biopsy

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Neuroblastoma mainly affects toddlers and young children – with 1,500 new cases per year in the EU region. Neuroblastoma is a malignant tumor of the peripheral nervous system and approximately 50% of patients are high-risk. Relapses occur frequently, and conventional therapy is no longer effective for these children. With liquid biopsies it is possible to monitor the success of therapy and to predict tumor recurrence in time to prevent treatment. Scientists from leading European research institutions in pediatric oncology are testing this promising diagnostic tool in coordination with the European Society for Pediatric Oncology (SIOPE) and under the scientific leadership of the Princess Maxima Center for Pediatric Oncology and St. Petersburg. Anna Children’s Cancer Research Institute. The framework is a five-year Horizon Europe project.

Liquid biopsies hold promise for the future of personalized cancer medicine: Many children with high-risk neuroblastoma, i.e. 50 percent of all neuroblastoma cases, do not respond to therapy and are at risk of recurrence. Until now, the success of the therapy has been monitored by medical imaging procedures and bone marrow tests. These tests are expensive, invasive, and stressful for children who require anesthesia. A liquid biopsy is a small blood sample, so the procedure is minimally invasive, and relatively simple. In the laboratory, samples can be used to determine whether children have responded to therapy. Finally, an impending recurrence can be detected early. Blood plasma is examined for genetic tumor markers, small fragments of DNA and messenger RNA (mRNA), which are secreted by tumor cells and provide information about genetic changes in the tumor. In cases of relapse, these may help 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 a defect in the ALK-gene, it can be targeted with drugs that inhibit ALK.

Liquid biopsy in clinical settings 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 benefits of this new diagnostic method – small blood samples from 150 patients with high-risk neuroblastoma will be collected several times and tested in laboratories such as the Princess Maxima Center and Labdia Laboradiagnostic GmbH. The European Society for Pediatric Oncology (SIOP Europe or SIOPE) is coordinating the research project, with a total funding of eight million euros. The project with the title SIOPEN Study to Monitor Neuroblastoma Relapse with Liquid Biopsy Sensitive Analysis – for short: Mona Lisa – aims to create a clinical study that can be quickly implemented in clinical practice.

“And this is already the first special feature,” said principal investigator Sabine Tasner-Mandl of St. Petersburg’s Anna Children’s Cancer Research Institute, who co-leads Monalisa’s liquid biopsy diagnostics. “We are using liquid biopsies in children with neuroblastoma for the first time in a clinical setting; so far, we have only tested them in studies,” said the tumor biologist.

Dr. Liv Tietgat, neuroblastoma specialist pediatric oncologist at the Princess Máxima Center for Pediatric Oncology and scientific co-head of the Mona Lisa project, said: ‘This study is a real breakthrough in the world of non-invasive diagnostics. Liquid biopsy is increasingly used in adults with cancer. Through this study we aim to be familiar with research in adults; An important development for children with cancer.’

Monitoring response to therapy and disease course is crucial to improve survival in high-risk neuroblastoma patients. Monalisa aims to close existing gaps in diagnostics and establish liquid biopsy as a standard method for monitoring relapsed neuroblastoma. The approach may also serve as a blueprint for other pediatric cancers. Marie Bernkopf, head of the Department of Diagnostics at Anna Children’s Cancer Research Institute and Labdia Laboratory in St. Petersburg, where the analyzes are performed, emphasizes that technological advances here will bring significant improvements for children and parents. For example, in some countries, mobile nurses will be able to take blood samples at home for check-ups, thus eliminating the burden of traveling to clinics.

Monalisa aims to analyze how patients and parents experience using liquid biopsy to adapt the procedure in the future. Through the project, scientists are taking a step toward effective personalized medicine for children with neuroblastoma and offer hope for a better chance of recovery.

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