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 23 035

Opportunity Information: Apply for PAR 23 035

This NIH funding opportunity (PAR-23-035) sits under the Gabriella Miller Kids First Pediatric Research Program and is designed to grow the Kids First Data Resource by generating high-quality genomic (and related) datasets from existing pediatric cohorts. The core idea is straightforward: investigators who already have well-characterized collections of samples from children with cancer and/or structural birth defects can apply to have those samples sequenced at a Kids First Program-supported sequencing center, with the resulting data and linked clinical or phenotypic information deposited for broad use by the research community. The notice is labeled X01 and "Clinical Trial Not Allowed," which signals that it is not meant to fund interventional clinical studies; instead, it supports research services and data generation tied to pre-existing cohorts and specimens.

Scientifically, the program is looking for cohorts that can shed light on the genetic basis of childhood cancers and structural birth defects, including both germline and somatic contributions where appropriate. Applications can focus on identifying inherited variants that predispose to cancer, tumor-specific (somatic) changes that drive pediatric malignancies, genetic causes and mechanisms underlying congenital anomalies, or the biological links between birth defects and later increased cancer risk. Another explicit goal is to broaden the set of pediatric conditions represented in the Kids First ecosystem, so proposals that bring in new disorders, new populations, or new types of pediatric datasets are aligned with the program's intent.

In terms of data types, whole genome sequencing is the main emphasis, but the announcement makes room for additional or alternative approaches when they are scientifically justified. In practice, that includes whole exome sequencing, transcriptome sequencing (for gene expression and fusion detection, for example), and epigenomic assays performed on tumor tissue or other affected tissues. The flexibility here is important: different pediatric diseases and sample types are better matched to different assays, and the program is signaling that it will consider the best-fit strategy as long as the rationale is clear and the cohort is appropriate.

A major cross-cutting priority is improving racial and ethnic representation in pediatric genomic resources. The announcement specifically encourages applications that focus on cohorts from underrepresented racial and ethnic groups or that measurably increase representation within projects already associated with Kids First. This reflects a broader concern in human genetics: datasets dominated by a narrow set of ancestries limit discovery, reduce the accuracy and portability of risk interpretation, and can widen disparities in downstream clinical translation. Proposals that address these gaps by design, rather than as an afterthought, are particularly responsive to the call.

The expected outcome of an awarded project is not just sequencing for a single lab's use, but a curated, shareable resource. Generated sequence data, along with associated clinical and phenotypic descriptors, will be incorporated into the Kids First Data Resource Center for access by the wider research community. That emphasis on structured data sharing is central to the program: it enables secondary analyses, cross-cohort comparisons, rare disease and rare cancer gene discovery through aggregation, and method development that would be hard to do with isolated, siloed datasets.

Eligibility is broad and includes many organization types that commonly hold pediatric cohorts and biospecimen collections. In addition to standard applicants such as universities, nonprofits, hospitals, small businesses, and various levels of U.S. government entities, the opportunity explicitly calls out a range of mission-driven and community-linked institutions. This includes Historically Black Colleges and Universities, Hispanic-serving institutions, Tribally Controlled Colleges and Universities, Alaska Native and Native Hawaiian Serving Institutions, and Asian American Native American Pacific Islander Serving Institutions, as well as faith-based or community-based organizations, U.S. territories or possessions, regional organizations, and non-U.S. (foreign) entities. The wide eligibility net fits the program's objective of capturing diverse cohorts wherever they exist, including outside traditional academic medical centers and outside the continental United States.

Administratively, the opportunity is a discretionary NIH grant in the health category (CFDA 93.310), with an original closing date listed as March 21, 2023, and the posting created February 6, 2023. The public summary does not state an award ceiling or expected number of awards in the provided fields, but the functional "award" in an X01 context is typically access to centralized sequencing and associated data generation pipelines rather than a large, flexible research budget. Applicants should therefore think in terms of proposing a well-defined cohort, strong scientific questions that sequencing can answer, clear justification for the assay strategy, and readiness to provide samples and metadata in a way that supports harmonization and downstream sharing through Kids First.

• 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 2023-02-06.
• Applicants must submit their applications by 2023-03-21. (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.

Apply for PAR 23 035

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