Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects: Gabriella Miller Kids First Pediatric Research Program (X01 Clinical Trial Not Allowed) | PAR 24 082

Opportunity Information: Apply for PAR 24 082

This NIH funding opportunity (PAR-24-082) supports the Gabriella Miller Kids First Pediatric Research Program, which is focused on uncovering the genetic drivers of childhood cancers and structural birth defects and making those data broadly available to the research community. Rather than funding a traditional hypothesis-driven clinical study, the emphasis here is on enabling high-quality genomic (and related) data generation from existing pediatric cohorts by having investigators submit biospecimens for sequencing through a Kids First Program-supported sequencing center. The overarching goal is to deepen understanding of how germline and/or somatic genetic variation contributes to pediatric cancer, to clarify the genetic causes of structural birth defects, and to explore the biological links that may explain why certain birth defects are associated with elevated cancer risk later in childhood. A key programmatic aim is also to broaden the scope of conditions represented in the Kids First Data Resource so that more pediatric disorders and more diverse patient populations are included.

Applicants are encouraged to propose sequencing on well-characterized cohorts that already exist, such as previously collected samples from children with cancer, children with congenital anomalies, or cohorts that capture both (for example, children with a specific birth defect who later develop malignancy). While whole genome sequencing is a central focus, the program is not limited to that approach. It can also support whole exome sequencing, transcriptome sequencing (RNA-seq), and, when scientifically justified, epigenomic assays performed on tumor tissue or other affected tissues. This flexibility is intended to let investigators match the assay strategy to the biology and sample types available, such as using tumor-normal paired sequencing to study somatic drivers in pediatric cancers, germline sequencing to identify inherited risk factors, transcriptomics to evaluate pathway disruption and gene expression signatures, or epigenomic profiling to capture regulatory changes that may be especially relevant in developmentally driven disorders.

A major expectation is that the generated genomic data will not remain isolated within a single lab or institution. Instead, the resulting sequence data, along with associated clinical and phenotypic information, will be incorporated into the Kids First Data Resource Center to enable responsible sharing with qualified researchers. In practical terms, applicants should be prepared to contribute well-annotated metadata describing the cohort, diagnoses, relevant clinical variables, and phenotypic descriptors so the data can be reused effectively. The program is designed to build a large, harmonized resource that supports secondary analyses, cross-cohort comparisons, discovery of rare variant associations, and improved understanding of pediatric disease mechanisms.

The announcement also places clear value on improving representation in pediatric genomics. Investigators are specifically encouraged to propose cohorts that include underrepresented racial and ethnic groups or to increase the diversity of datasets already in the Kids First ecosystem. This reflects a broader NIH priority to reduce disparities in genomic research, improve the generalizability of discoveries, and avoid creating reference resources that primarily reflect limited ancestry groups.

The award mechanism is an X01 (clinical trial not allowed), which typically signals that the main value provided is access to a program resource or service rather than a large, flexible pot of direct research dollars. In this case, that resource is sequencing and associated data generation through Kids First-supported sequencing centers, with the downstream expectation of data deposition and sharing through the Kids First Data Resource. Because clinical trials are not allowed, applications should not propose prospective interventional studies that assign participants to interventions to evaluate health outcomes. The focus is on sequencing and data generation from pediatric cohorts, generally leveraging existing samples and clinical information.

Eligibility is broad and includes many types of organizations that may hold relevant pediatric cohorts or have the partnerships needed to assemble them. Eligible applicants listed include state, county, city, and special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; nonprofit organizations (with or without 501(c)(3) status); for-profit organizations (other than small businesses) and small businesses; public housing authorities/Indian housing authorities; and Native American tribal governments and tribal organizations (including federally recognized tribes and other tribal entities). The opportunity explicitly highlights additional eligible groups such as Historically Black Colleges and Universities (HBCUs), Hispanic-serving Institutions, Tribally Controlled Colleges and Universities (TCCUs), Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), faith-based or community-based organizations, non-U.S. entities (foreign organizations), regional organizations, and U.S. territories or possessions. This wide eligibility is consistent with the program goal of capturing diverse cohorts and supporting contributions from institutions that serve populations historically underrepresented in large-scale genomic datasets.

From an administrative standpoint, the opportunity is issued by the National Institutes of Health under CFDA (Assistance Listing) 93.310, categorized under health, using a grant funding instrument. The original closing date listed is March 13, 2024, and the opportunity was created on December 12, 2023. The posted award ceiling and expected awards fields are not specified in the provided excerpt, which is consistent with service-based mechanisms where the “award” is often tied to sequencing capacity and program priorities rather than a simple per-award dollar cap.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects: Gabriella Miller Kids First Pediatric Research Program (X01 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.310.
• This funding opportunity was created on 2023-12-12.
• Applicants must submit their applications by 2024-03-13. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

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