Summary
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).
Eligibility
- Independent School Districts
- State Governments
- Nonprofits Non Higher Education Without 501c3
- Native American Tribal Governments Federally Recognized
- County Governments
- Public and State Institutions of Higher Education
- For Profit Organizations Other Than Small Businesses
- Native American Tribal Organizations Other Than Federally Recognized Tribal Governments
- Small Businesses
- Nonprofits Having A 501(c)(3) Status With the Irs Other Than Institutions of Higher Education
- Public and Indian Housing Authorities
- Special District Governments
- Private Institutions of Higher Education
- City Or Township Governments
Funding categories
- Agriculture
- Consumer Protection
- Food and Nutrition
Official source and attribution
This directory republishes federal opportunity information sourced from Simpler.Grants.gov and Grants.gov. Local Business Ranks is not affiliated with or endorsed by the federal government. Always rely on the official opportunity record for application requirements and changes.
Open the official federal record