Related Experiment Video
Updated: Jun 27, 2026
Proton Therapy Delivery and Its Clinical Application in Select Solid Tumor Malignancies
Published on: February 6, 2019
Precision neuro-oncology for children: Time to gear up!
Julie Lafont1, Mathilde Kerhervé1, Nicolas André2
1CNRS, INSERM, Aix Marseille Univ, Institut Paoli Calmettes, Centre de Recherche en Cancérologie de Marseille (CRCM), REMAP4Kids Team, Equipe Labélisée par la Ligue Nationale Contre le Cancer, SouthROCK Center of Excellence in Pediatric Oncology Research, Marseille, France.
Abstract:
Molecular profiling is central to pediatric precision oncology, yet only 15-20% of patients benefit from biology-guided treatments. This limitation underscores the urgent need for functional precision medicine, which interrogates therapeutic vulnerabilities using patient-derived models. Despite hurdles in establishing these models, such as limited biopsy material and technical screening barriers, recent advances in tumoroids, patient-derived xenografts and high-throughput platforms offer an unprecedented opportunity to bridge the translational gap. In this review, we describe the evolution of patient-derived models of pediatric brain cancers, their current strengths and weaknesses and the technological innovations redefining the field. Together, these insights provide a road map for integrating functional data into clinical decision-making, ultimately aiming to 'cure more and heal better' children with brain tumors.
Insights
Functional precision medicine using patient-derived models is crucial for pediatric brain cancer, as molecular profiling offers limited benefits. Advances in models like tumoroids and xenografts are key to improving treatment outcomes for children.
Area of Science:
- Pediatric Oncology
- Translational Medicine
- Cancer Research
Background:
- Molecular profiling benefits only 15-20% of pediatric cancer patients.
- There is a critical need for functional precision medicine to identify effective treatments.
- Patient-derived models are essential for interrogating therapeutic vulnerabilities.
Purpose of the Study:
- To review the evolution of patient-derived models for pediatric brain cancers.
- To discuss the strengths and weaknesses of current models.
- To highlight technological innovations in the field.
Main Methods:
- Review of scientific literature on patient-derived models.
- Analysis of advances in tumoroids, patient-derived xenografts, and high-throughput platforms.
- Discussion of challenges in establishing and utilizing these models.
Main Results:
- Patient-derived models, including tumoroids and xenografts, show promise in bridging the translational gap.
- Technological innovations are overcoming previous hurdles like limited biopsy material.
- These models offer a pathway to functional precision medicine.
Conclusions:
- Patient-derived models are vital for advancing pediatric precision oncology.
- Integrating functional data from these models can improve clinical decision-making.
- The ultimate goal is to enhance treatment efficacy and outcomes for children with brain tumors.
Related Concept Videos
02:56Treatment Resistant Cancers
02:56Treatment Resistent Cancers