Abstract 1879: A novel therapeutics accelerator for the brain cancer community: Early lessons learned

Abstract Developing a comprehensive translational program for brain tumors is a complex task that involves numerous challenges. These challenges include significant obstacles surrounding the delivery of pre-clinical studies, safety, clinical trial design, and routes to commercialization. Recognizing that this journey is difficult, in 2023 the Tessa Jowell Brain Cancer Mission launched the Brain Tumor Research Novel Therapeutics Accelerator (BTR-NTA). The BTR-NTA is a new international accelerator program that provides independent and comprehensive expert guidance to de-risk therapeutic development through systematic, multidisciplinary evaluation and feedback for academic or industry researchers who are developing a therapeutic or technology for brain tumors. The BTR-NTA was modeled on the TREAT-NMD Advisory Committee for Therapeutics, which has yielded impressive results in the neuromuscular disease field. The ultimate goal of the BTR-NTA program is to enable researchers with a potential therapy or technology for brain tumors to navigate the development journey and successfully make it to the clinic in an efficient and timely way. The international, multidisciplinary BTR-NTA Expert Committee spans the entire therapeutic development pathway to avoid a siloed approach to research. Thanks to charitable funding, the BTR-NTA is free for academics and available at low cost for industry. Researchers at any stage of pre-clinical or clinical work can apply to the BTR-NTA, and, if accepted, their application will proceed as follows: 1. The applicant completes an application form covering work to date and future plans under a strict confidentiality agreement that protects the applicant; 2. Depending on the types of applicants, additional experts are recruited to ensure that each applicant gets a robust and tailored review 3. The applicant’s work is reviewed by an international, multidisciplinary committee 4. The applicant and BTR-NTA Expert Committee meet in-person to further discuss the applicant’s work; and 5. The applicant receives a comprehensive written report from the BTR-NTA Expert Committee, with feedback on strengths and weaknesses and guidance on next steps. The BTR-NTA Expert Committee dedicates half a day to review the applicant’s work in-person, and extensive time pre- and post-meeting. This is a low cost to applicant, high impact process designed to provide valuable feedback and support. The BTR-NTA aims to help overcome barriers for applicants by highlighting potential pitfalls or risks, providing multi-disciplinary feedback on work to date and constructive guidance on next steps, and by strengthening international collaboration in the brain tumor field. Following the launch of the BTR-NTA program in 2023 and review of the first group of therapeutics and technologies, we report on our early learnings, impact, and how we hope to grow the program to help accelerate new brain tumor therapies globally. Citation Format: Charlotte Kate Aitken, Katie Bushby, Petra Hamerlik, Nicky Huskens, Edward Kaye, Dione Kobayashi, Juanita Lopez, Ryan Mathew, John McCall, Javad Nazarian, Ruth Plummer, Steven Pollard, Ruman Rahman. A novel therapeutics accelerator for the brain cancer community: Early lessons learned abstract. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts) ; 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84 (6Suppl): Abstract nr 1879.