OpenResearch Grant

Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers

Funded by:

Food and Drug Administration

Grant Amount

USD: 1,500,000Program Funding

USD: 500,000Award Minimum

Deadline

Jun 15, 2026

4 days remaining

Est. Awards

3 Grants

Funding Purpose

Applied ResearchInnovation Grants

Subjects

MedicinePharmacology, Toxicology and Pharmaceutics

Description

Overview

The Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers opportunity, issued by the Food and Drug Administration (FDA) through the Oncology Center of Excellence (OCE), seeks to advance the development and regulation of oncology products for patients with ultra-rare cancers. This notice of funding opportunity (RFA-FD-26-004) supports 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. For this purpose, ultra-rare cancers are defined as those with an approximate annual incidence in the United States of 300 to 400 people or less. The OCE’s Pediatric Oncology Program, Rare Cancers Program, and Project Catalyst, along with the public-private partnership Ultra-Rare Cancer Treatment Advancement Program (ULTRA), provide the broader context for this initiative.

Research Areas

Specific areas of interest include, but are not limited to:

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, 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).

Funding and Benefits

Program Funding: $1,500,000

Expected Number of Awards: 3

Award Maximum: $500,000

Funding Instrument Type: Cooperative Agreement

Cost Sharing or Matching Requirement: No

Category of Funding Activity: Agriculture; Consumer Protection; Food and Nutrition

Eligibility

Eligible applicants include:

Education: Private institutions of higher education; Public and state institutions of higher education; Independent school districts.
Government: Special district governments; County governments; Federally recognized Native American tribal governments; City or township governments; Public and Indian housing authorities; State governments.
Nonprofit: Other Native American tribal organizations; Nonprofits non-higher education without 501(c)(3); Nonprofits non-higher education with 501(c)(3).
Business: Small businesses; For-profit organizations other than small businesses.

Application Process

Applications must be submitted through Grants.gov. Visit Grants.gov to apply, track application status, and subscribe to updates. The funding opportunity number is RFA-FD-26-004.