Personalized Targeted Therapy for Refractory Pediatric Brain Tumors Program: Concept and Preliminary Clinical Experience

As former diagnoses are separated in smaller groups, many novel agents will never be studied for pediatric malignancies with traditional clinical research methods. In clinical care of refractory pediatric brain tumors, we have recently personalized the treatment by adding agents based on tumor markers. The algorithm starts with the treatment history and evidence-based drug rankings, and creating a first personalized treatment cycle. In addition, each tumor is analyzed for a panel of markers resulting in novel drugs to be added to the second cycle. Here we report a preliminary retrospective chart review of patien…