Working Together to Accelerate the Preclinical to Clinical Translation of Drug Delivery Systems for Children’s Brain Tumours

Abstract AIMS Several techniques, such as intra-cerebrospinal fluid chemotherapy, ultrasound-mediated blood-brain barrier disruption, convection enhanced delivery, polymer delivery systems, electric field therapy, and intra-arterial intra-nasal have the potential to transform treatment of brain tumours in children. However, there been very few clinical trials evaluate these. We aim address challenges pre-clinical research pathway for CNS drug systems order increase number this area. METHOD In 2021, CBTDDC (Children’s Brain Tumour Drug Delivery Consortium) ITCC (Innovative Therapies Children with Cancer) tumour group established a Clinical Trials Working Group comprising international researchers clinicians issue. RESULTS This partnership highlighted main as: (1) lack specific funding prototype development and/or scale-up trials; (2) difficulties navigating regulatory landscape; (3) accurate preclinical models; (4) increased need multi-centric working. response this, we ran workshop on ‘Clinical Trial Readiness’, attended by around 50 delegates (comprising clinicians, researchers, trial experts, policy makers, representatives from organisations, charities industry). CONCLUSION now speciality-specific working groups producing recommendations use models techniques according type. also created ‘Roadmap’ document translation, which will be freely shared neuro-oncology community.