Based on the hypothesis that the PDGFRA oncogene, which is frequently present in pediatric brain tumors [13,14,47], may also be involved in sensitization to DNA damaging agents [17], we explored in vivo the combination of regorafenib with a topoisomerase I inhibitor and irradiation, both treatments commonly used for brain tumors in patients. This evidence concerns the gene PDGFRA and brain neoplasm.