Abstract B010: Common translational challenges in brain tumour therapeutic development identified through a multidisciplinary accelerator programme

Abstract The purpose of this thematic analysis was to synthesise key translational challenges identified through the Brain Tumour Research Novel Therapeutics Accelerator (BTR-NTA), an international, multidisciplinary programme established to support the development of novel brain tumour therapies and accelerate their progression towards clinical application through structured expert input. Between 2023 to 2025, BTR-NTA received 33 applications and provided up to 240 hours of tailored input – from experts in preclinical, clinical, regulatory, commercialisation and patient advocacy domains - for 15 academic and industry groups developing novel brain tumour therapies. A thematic analysis was then conducted across these 15 therapeutic development programmes reviewed within the accelerator, using the structured feedback reports provided to applicants to identify recurring development challenges. The analysis identified common challenges across all major stages of therapeutic development, for which the Committee provided input and guidance. 93% of programmes were provided with input to refine their preclinical strategies, including the use of more clinically relevant disease models and potential collaboration partners. Similar proportions (87%) exhibited gaps in pharmacology and toxicology planning that posed potential risks to progression towards first-in-human studies. Clinical development planning input was provided to 100% of programmes, including recommendations on patient selection criteria, dosing schedule and biomarker selection. Regulatory engagement strategies were also provided for 87% of programmes, most commonly relating to the timing and nature of regulatory engagement and anticipated data requirements. Finally, 93% of applicants received guidance on their patient and public involvement and engagement strategy. Follow-up with applicants one year after their BTR-NTA review is beginning to demonstrate the long-term impact of this guidance, with 100% of applicants who responded reporting they had adapted an aspect of their development strategy based on advice from their BTR-NTA review, and 100% reporting receiving follow on funding from government, charitable and venture capital sources. These findings demonstrate that scientifically promising therapeutic programmes consistently faced a number of translational challenges that could delay or prevent successful clinical progression if left unaddressed. The primary objective of the BTR-NTA programme is to identify these risks early and to provide targeted, practical solutions. Wider dissemination of these learnings is hoped to improve translational efficiency and accelerate progress towards effective new treatments for people with brain tumours. Citation Format: Eloise Lines, Charlotte Aitken, Katie Bushby, Nicky Huskens, Karen Noble, Paul Brennan, Helen Bulbeck, Petra Hamerlik, Darren Hargrave, Edward Kaye, Dione Kobayashi, Ryan Mathew, Javad Nazarian, Ruman Rahman, Artie Romero, Erik Sulman, Juanita Lopez, Kanneboyina Nagaraju. Common translational challenges in brain tumour therapeutic development identified through a multidisciplinary accelerator programme [abstract]. In: Proceedings of the AACR Special Conference in Cancer Research: Brain Cancer; 2026 Mar 23-25; Philadelphia, PA. Philadelphia (PA): AACR; Cancer Res 2026;86(6_Suppl):Abstract nr B010.