Abstract A034: A new international program to accelerate novel therapeutics for brain tumors: presenting the brain tumor research novel therapeutics accelerator

Abstract Each year, we see many journal manuscripts highlighting successful pre-clinical experiments for potential new brain tumor therapies. Only a few of these therapies move forward into successful clinical trials. Indeed, developing a comprehensive translational program covers many aspects, including but not limited to, medicinal chemistry, blood brain barrier penetration, tumor heterogeneity, delivery, commercialization, and navigating the regulatory environment. Because of this, bringing a successful translational project from bench to bedside requires access to a diverse set of cutting-edge expertise to give promising therapies the best chance of succeeding. Recognizing that this journey is not easy to navigate, the Tessa Jowell Brain Cancer Mission has launched an international Brain Tumor Research Novel Therapeutics Accelerator (BTR-NTA). This program is led by an international group of drug development and brain tumor experts who provide independent guidance to researchers to position their therapeutic along a realistic pathway to clinical trials. The BTR-NTA is open to any international academic and industry researchers developing a medicinal product, delivery technology or device for brain tumors. The BTR-NTA welcomes groups at any stage of pre-clinical or clinical work. Academics and smaller biotech’s could benefit from feedback on their pre-clinical studies and plans for commercialization, while larger pharmaceutical companies could benefit from access to brain tumor specific expertise and patient input into clinical trial design. Researchers can apply to BTR-NTA twice a year, and if accepted will go through the following process: 1. Complete an application form covering work to date and future plans under strict confidentiality agreements; 2. Attend an in-person meeting with a tailored group of experts to further discuss their work; and 3. Receive a comprehensive written report from the BTR-NTA committee with feedback on strengths and weaknesses and guidance on next steps. The expert committee dedicates half a day to reviewing the applicants’ work in-person, and extensive time pre- and post-meeting. Throughout the process, applicants receive up to 150 hours of expert input. The multidisciplinary nature of the review committee spans the entirety of the drug development pathway to support longitudinal collaboration and avoid a siloed approach to research. The BTR-NTA was designed after a similar program in another rare disease field yielded impressive results. Thanks to charitable funding, the BTR-NTA is free to academics and at low cost for industry. The BTR-NTA aims to help address barriers for researchers by highlighting potential pitfalls or risks, providing multi-disciplinary feedback on work to date, giving constructive guidance on next steps and strengthening international collaboration in the brain tumor field. The ultimate goal of the BTR-NTA is to enable researchers with therapy for brain tumors to navigate the drug development journey and make it to the clinic in a low-cost and timely way. Citation Format: Nicky Huskens, Charlotte Aitken, Katie Bushby. A new international program to accelerate novel therapeutics for brain tumors: presenting the brain tumor research novel therapeutics accelerator abstract. In: Proceedings of the AACR Special Conference on Brain Cancer; 2023 Oct 19-22; Minneapolis, Minnesota. Philadelphia (PA): AACR; Cancer Res 2024;84 (5 Suppl₁): Abstract nr A034.