Discovery of the Genetic Basis of Childhood Cancers and of Congenital Anomalies: Gabriella Miller Kids First Pediatric Research Program (X01 Clinical Trial Not Allowed)

The Discovery of the Genetic Basis of Childhood Cancers and of Congenital Anomalies program is a federal funding opportunity administered by the National Institutes of Health through the Office of Strategic Coordination within the Common Fund. It operates under the Gabriella Miller Kids First Pediatric Research Program, which was established to accelerate discoveries in childhood cancer and congenital anomalies by leveraging large-scale genomic data. The program is managed collaboratively across NIH institutes, with leadership from the National Cancer Institute and the Eunice Kennedy Shriver National Institute of Child Health and Human Development. Its overarching mission is to enable researchers to uncover genetic mechanisms underlying pediatric diseases and to translate those findings into improved diagnostics, prevention strategies, and treatments. The purpose of this opportunity is to provide investigators with access to genomic sequencing resources rather than direct monetary funding. Applicants propose cohorts of pediatric patients with childhood cancers or congenital anomalies and submit biological samples for sequencing through NIH-supported data generating centers. The program supports whole genome, exome, transcriptomic, epigenomic, and other omics-based analyses, including long-read sequencing and proteomics where justified. The resulting genomic and clinical data are integrated into the Kids First Data Resource Center, a shared platform designed to facilitate broad access and collaboration across the research community. Funding under this mechanism does not provide direct financial awards to recipients. Instead, it offers access to high-cost sequencing infrastructure and associated data processing capabilities. As such, applicants cannot request funds for sample collection, additional phenotyping, or data analysis. All sequencing is performed by designated centers, and investigators must collaborate with these centers and NIH staff to finalize project design and sample processing. The project period is limited to one year, with expectations that samples be shipped within six months of award notification. This structure emphasizes rapid data generation and integration into shared resources rather than traditional grant-funded research activities. Eligibility for this opportunity is broad and includes U.S.-based higher education institutions, nonprofits, for-profit organizations, and various government entities at the state, local, and tribal levels. However, foreign organizations and non-domestic components of U.S. institutions are not eligible to apply. Applicants must demonstrate that they have high-quality samples ready for sequencing and that participants have provided consent for broad data sharing. Cohorts with rich clinical and phenotypic data and those that enhance diversity or fill gaps in existing datasets are prioritized. The program encourages collaborative approaches, including pooling samples across investigators to increase statistical power and representation. The application process requires submission through Grants.gov using NIH systems such as ASSIST or institutional system-to-system solutions. Applicants must follow the NIH Research (R) Application Guide and include required components such as Institutional Certification for genomic data sharing, detailed sample inventories, and clinical data descriptions. Applications are due by January 11, 2027, with an earliest submission date of December 11, 2026. Scientific merit review is scheduled for March 2027, and the earliest anticipated project start date is April 2027. Evaluation is conducted by the Gabriella Miller Kids First Working Group, focusing on the scientific merit of the proposed cohorts, the likelihood of uncovering meaningful genetic insights, and the contribution to the broader data resource. Reviewers assess factors such as study design, sample quality, data sharing compliance, and potential impact on pediatric research. Selected projects are expected to actively contribute to the Kids First Data Resource and participate in collaborative efforts to maximize the utility of the generated data. This opportunity is part of an ongoing federal initiative and represents a continuation of prior funding announcements, indicating a recurring program structure.

Prioritize high-quality samples ready for sequencing; ensure broad data sharing consent; include rich clinical data; demonstrate strong scientific rationale and cohort value