Abstract PR005: Helping academic investigators develop small molecules for the clinic: The NCI Developmental Therapeutics Program and Stepping-Stones
Authors: Verma Sharad , Hayre Morgan O' , Aurigemma Rose 
Published: December 2024

Abstract
Academic researchers working in small molecule drug discovery and development face unique challenges – key among these include limited access to the full range of resources needed, inadequate funding which does not cover costs for routine/iterative product development tasks, and unavailability of expertise for the full range of regulatory critical path steps required for an IND. For academic institutions which lack deep resources, specialized core facilities, or endowments, these challenges are even more dire. In recognition of these realities, the NCI Developmental Therapeutics Program (DTP) expanded its capability through creation of the Stepping-Stones program, which provides resources to academic-based researchers who are working towards advancing their small molecule therapeutic candidates from ‘bench to bedside’. Through the Stepping-Stones program, NCI grant-supported small molecule therapeutics focused projects demonstrating high productivity are eligible to obtain access to NCI/DTP drug development capabilities and resources to fill in ‘gaps’ for specific activities that are not covered through their existing funding, but which are necessary to facilitate advancement along the critical path. Examples of services provided have included molecular profiling for ADME/DMPK studies, screens for secondary pharmacology and in vitro safety, synthesis optimization and chemical synthesis, and formulation development, to name a few. By filling in such discrete ‘gaps’, project teams can ‘de-risk’ a candidate earlier and develop stronger data packages to better position themselves for attracting additional investment for development toward clinical testing, or as the data warrants pursue an earlier re-direction of effort before going too far down the critical path. An overview of Stepping-Stones including details for how to be considered for this program and its interactions with other programs at NCI that support drug discovery and development is described, in addition to an overview of DTP’s discovery and development services which usher R&D programs from discovery through preclinical development and IND-enabling studies to support first-in-human trials. From this presentation, academic based ‘drug hunters’ will obtain knowledge about the Stepping-Stones program as a support mechanism to enable their continued efforts and gain an improved understanding about the ‘ecosystem’ of drug discovery and development programs offered by the NCI for the extramural community of researchers.
Citation Format: Sharad Verma, Morgan O' Hayre, Rose Aurigemma. Helping academic investigators develop small molecules for the clinic: The NCI Developmental Therapeutics Program and Stepping-Stones [abstract]. In: Proceedings of the AACR Special Conference in Cancer Research: Optimizing Therapeutic Efficacy and Tolerability through Cancer Chemistry; 2024 Dec 9-11; Toronto, Ontario, Canada. Philadelphia (PA): AACR; Mol Cancer Ther 2024;23(12_Suppl):Abstract nr PR005