Novel approaches to support therapeutic development in ultra-rare cancers

id: 360223
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opportunityNumber: FOR-FD-26-004
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opportunityTitle: Novel approaches to support therapeutic development in ultra-rare cancers
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revision: 1
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agencyDetails: {"code":"FDA","seed":"HHS-FDA","agencyName":"Food and Drug Administration","agencyCode":"HHS-FDA","topAgencyCode":"HHS"}
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owningAgencyCode: HHS-FDA
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cfdas: [{"id":430460,"opportunityId":360223,"cfdaNumber":"93.103","programTitle":"Food and Drug Administration Research"}]
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forecastDesc: <p>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.&nbsp;</p><p><br></p><p>Specific areas of interest include, but are not limited to, the following examples:</p><p>• 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.</p><p>• Investigations to explore opportunities to develop and validate early clinical endpoints and other novel efficacy endpoints for evaluation of treatments for ultra-rare cancers.</p><p>• 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).&nbsp;</p><p>• 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.</p><p>• 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</p><p>• Development of methods to incorporate use of telemedicine and/or pragmatic trial design elements&nbsp;(e.g., collecting laboratory and/or imaging data from local facilities) for patient assessments to facilitate enrollment of patients with ultra-rare cancers</p><p>• 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.</p><p>• 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).</p><p><br></p>
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applicantTypes: [{"id":"13","description":"Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education"},{"id":"04","description":"Special district governments"},{"id":"20","description":"Private institutions of higher education"},{"id":"22","description":"For profit organizations other than small businesses"},{"id":"06","description":"Public and State controlled institutions of higher education"},{"id":"08","description":"Public housing authorities/Indian housing authorities"},{"id":"01","description":"County governments"},{"id":"11","description":"Native American tribal organizations (other than Federally recognized tribal governments)"},{"id":"00","description":"State governments"},{"id":"07","description":"Native American tribal governments (Federally recognized)"},{"id":"23","description":"Small businesses"},{"id":"02","description":"City or township governments"},{"id":"12","description":"Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education"},{"id":"05","description":"Independent school districts"}]
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fundingInstruments: [{"id":"CA","description":"Cooperative Agreement"}]
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fundingActivityCategories: [{"id":"AG","description":"Agriculture"},{"id":"CP","description":"Consumer Protection"},{"id":"FN","description":"Food and Nutrition"}]
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costSharing: false
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lastUpdatedDate: Jan 08, 2026 05:04:04 PM EST
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status: forecasted
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