Clinical Research Directory

Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma

The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in the pilot phase of performing real-time drug screening on tissue taken during surgery in patients with relapsed medulloblastoma or ependymoma and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed medulloblastoma and ependymoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer.

Multiomic Approach to Radioresistance of Ependymomas in Children and Adolescents

Treatment of childhood ependymoma, the second most frequent pediatric brain tumor, is based on surgery and radiation therapy. However, 50% relapse, mainly locally. Progress in imaging, molecular biology and radiotherapy ballistics has led us to propose the EPENDYMOMICS project, a multi-omics approach using artificial intelligence to detect the predictive characteristics of relapse, and to define innovative radiotherapy targets using multimodal imaging. We previously reported that the relapse sites are mainly located in the high-dose radiotherapy zone and that there appear to be prognostic factors for relapse based on anatomical and functional MRI abnormalities by diffusion and perfusion. In addition, recent studies in molecular biology have identified significant prognostic factors. The challenge now is to use and correlate all these findings in larger cohorts to tackle the radio-resistance of this disease. Our objective is to collate in a single database called NETSPARE (Network to Structure and Share Pediatric data to Accelerate Research on Ependymoma) the clinical, histological, biological, imaging and radiotherapy data from two consecutive studies that included 370 children and adolescents with ependymoma since 2000 in France. The EPENDYMOMICS project will comprise a clinical research team, three imaging research teams, two histopathology teams, and a biostatistics team working on NETSPARE. Our goal is to obtain a radiogenomic signature of our data, which will be validated with the English external cohort of 200 patients that is currently being analyzed. The perspective is to optimize the indications and volumes of irradiation that could in the future be used in a European translational research trial to tackle radioresistance.