New drugs are the cornerstone to improving survival among children with cancer. For high-grade glioma, only four drugs have been licensed in the past four decades; of which three are still in production (bevacizumab, lomustine, and carmustine). All were selected to overcome both the blood–brain barrier and resistance to chemotherapy and targeted therapies. Only one of these drugs, temozolomide, is licensed for paediatric high-grade glioma in some countries, despite few trials supporting its activity. 1 The situation is even worse in paediatric diffuse intrinsic pontine glioma (DIPG). Preclinical research on DIPG has identified several compounds with potential activity; 2 however, these agents need to be evaluated rigorously using state-of-theart methods and, if proven valuable, made available to all patients worldwide.