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

Abstract

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