Artificial Intelligence has helped researchers make major progress in treating an incurable childhood brain cancer. Image Pictured: Professor Chris Jones, Professor of Paediatric Brain Tumour Biology at the ICR The approach allowed researchers at The Institute of Cancer Research, London and The Royal Marsden to explore ideas on how to target DIPG in young patients who have mutations in the ACVR1 gene, which is about a quarter of children with this type of cancer. There is currently no treatment targeting this mutation approved for use in DIPG. The targeted drug vandetanib, which is approved for treating thyroid cancer, acts against the ACVR1 gene. However, it has difficulty getting past the blood-brain barrier, so it cannot build up to a high enough concentration in the brain to have a therapeutic effect. But by using an artificial intelligence (AI) drug-discovery platform built by BenevolentAI, scientists found that a drug called everolimus could prevent vandetanib from being ejected from the brain, allowing it to treat the cancer. Study leader Professor Chris Jones, Professor of Paediatric Brain Tumour Biology at the ICR, said: “DIPG is a rare and aggressive childhood brain cancer, and survival rates have not changed over the past 50 years, so we desperately need to find new treatments for this disease. “The AI system suggested using a combination of two existing drugs to treat some children with DIPG – one to target the ACVR1 mutation, and the other to sneak the first past the blood-brain barrier. The treatment extended survival when we tested it in a mouse model, and we have already started testing it out in a small number of children.” Dr Fernando Carceller, a Consultant Paediatric Oncologist at The Royal Marsden and Leader of the Paediatric & Adolescent Neuro-Oncology and Drug Development team at the ICR, said: “Close collaboration between scientists at the ICR and clinicians at The Royal Marsden made this bench-to-bedside approach possible. “The clinical results are too preliminary so as to draw firm conclusions, but we are working to take this combination forward in a clinical trial for children with ACVR1-mutant DIPG.” The research was published in the journal Cancer Discovery and was funded by Brain Research UK, the DIPG Collaborative, Children with Cancer UK, Abbie’s Army, Lucas’ Legacy, the Lyla Nsouli Foundation, Cancer Research UK, and a George and the Giant Pledge donation to The Royal Marsden Cancer Charity, with additional support from the NIHR Biomedical Research Centre at The Royal Marsden and the ICR.