Novel approaches to support therapeutic development in ultra-rare cancers
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This program aims to advance therapeutic development specifically for ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers. It focuses on novel and collaborative approaches that address critical challenges such as the creation of infrastructure for coordinated networks and data repositories to support drug development and regulatory processes. Key priorities include exploring and validating new clinical endpoints, utilizing real-world data through registries, particularly for pediatric diffuse midline glioma, and devising strategies to repurpose or preserve drugs that have been discontinued commercially but show promise in ultra-rare cancers. Additional emphasis is placed on innovative delivery methods like nanoparticle-based therapeutics targeting specific transcription factors, the use of telemedicine and pragmatic trial designs to ease patient enrollment, and comprehensive molecular profiling of ultra-rare tumors to identify unique therapeutic targets. The program encourages multi-stakeholder cooperation and cutting-edge research methodologies including single-cell transcriptomics and proteomics to better understand tumor biology and improve treatment outcomes. Overall, the initiative seeks to overcome limitations in drug development driven by rarity and financial disincentives while fostering new scientific and clinical advances in this specialized field.
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The purpose of this program is to support new approaches that can be applied to facilitate therapeutic development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers.
Specific areas of interest include, but are not limited to, the following examples:
• Development of infrastructure for a coordination network and data repository for patient-level data across institutions and internationally to support drug development and regulatory decision-making for one or more ultra-rare cancers.
• Investigations to explore opportunities to develop and validate early clinical endpoints and other novel efficacy endpoints for evaluation of treatments for ultra-rare cancers.
• Development and implementation of a collaborative multi-stakeholder effort to support generation and use of real-world data leveraging a registry framework for use in development of new therapies for pediatric patients with diffuse midline glioma (DMG) (including diffuse intrinsic pontine glioma, DIPG).
• Innovative approaches to identify new biologically-driven opportunities for clinical development of previously approved drugs or biologics (hereafter referred to as drugs), including drugs for which development has been discontinued, in ultra-rare cancers.
• Research to develop novel approaches to preserve the availability of drugs for which commercial developers have discontinued adult development that have strong potential in ultra-rare cancers but lack financial incentives for commercial development
• Development of methods to incorporate use of telemedicine and/or pragmatic trial design elements (e.g., collecting laboratory and/or imaging data from local facilities) for patient assessments to facilitate enrollment of patients with ultra-rare cancers
• Development of nanoparticle-based delivery approaches for therapeutic nucleic acids targeting onco-fusion transcription factors in metastatic tumor animal models using targeted bioPROTAC degradation or genomic editing strategies. Successful efforts should demonstrate effective delivery and expression in-vivo to tumor cells, and downregulation of the target transcription factor protein while minimizing off-target effects and limiting sequestration of the nanoparticle by the liver, spleen, and lungs.
• Research to exhaustively characterize the plasma-membrane protein expression (surfaceome) of an ultra-rare cancer and the presumed healthy tissue of origin, as well as the resident-tissue stem cells, by single-cell transcriptomics and proteomics. These studies, and available correlative database analyses, should be designed to identify possible combinatorial signatures of plasma membrane proteins unique to the ultra-rare tumor. Tumors of interest include Sclerosing epithelioid fibrosarcoma and atypical teratoid rhabdoid tumors (ATRT).