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 21 040

Opportunity Information: Apply for PAR 21 040

The NIH funding opportunity PAR-21-040, 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)," supports the Gabriella Miller Kids First Pediatric Research Program (Kids First) by inviting research teams to contribute pediatric cohort biospecimens for sequencing through a Kids First-supported sequencing center. The central goal is to accelerate discovery of genetic factors that contribute to childhood cancers and to structural birth defects by generating high-quality genomic data from well-characterized pediatric cohorts and pairing those data with relevant clinical and phenotypic information. This is not a clinical trial mechanism, and it is structured around enabling data generation and broad data sharing for the wider pediatric research community.

A key emphasis of the opportunity is on leveraging existing cohorts. Applicants are encouraged to propose sequencing for previously assembled pediatric cancer cohorts to help clarify how inherited and acquired genetic variation influences cancer risk, tumor biology, and potentially outcomes. In parallel, applicants can propose cohorts focused on structural birth defects, with the aim of expanding the breadth of disorders represented in the Kids First Data Resource and improving understanding of the genetic etiology of congenital anomalies. The program is designed to build a shared resource rather than to fund traditional hypothesis-driven bench or interventional clinical studies, with the primary deliverable being sequence data and associated curated metadata that can be reused by many investigators.

The primary sequencing approach highlighted is whole genome sequencing, but the announcement also notes that whole exome and transcriptome sequencing may be available in situations where they are well justified, such as when tumor samples or other affected tissues are proposed and additional molecular profiling is needed to appropriately characterize the disease biology. This flexibility is important for pediatric cancer studies in particular, where paired tumor-normal sequencing and expression data can be crucial for distinguishing germline variation from somatic mutations and for interpreting disrupted pathways. Regardless of sequencing type, the expectation is that data generation will occur at a sequencing center supported by Kids First, helping standardize methods and quality across contributed cohorts.

A defining feature of this program is data sharing through the Gabriella Miller Kids First Pediatric Data Resource. The genomic data produced under this opportunity, along with associated clinical and phenotypic datasets, are intended to become part of the Kids First Data Resource and be made available to the broader pediatric research community. In practice, this means applicants should be prepared to supply not only samples but also robust, well-organized phenotype and clinical annotation so that downstream researchers can meaningfully analyze genotype-phenotype relationships. The overall value proposition is that a single contributed cohort can support many future studies, including cross-cohort analyses that increase statistical power for rare pediatric diseases and enable discovery of shared or distinct genetic mechanisms across conditions.

In terms of who can apply, eligibility is broad and includes many organization types across government, academia, nonprofit, and industry. Eligible applicants listed include state, county, city/township, and special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; Native American tribal governments (federally recognized); tribal organizations that are not federally recognized; public housing authorities/Indian housing authorities; nonprofits with and without 501(c)(3) status; for-profit organizations other than small businesses; small businesses; and other entities. The opportunity also explicitly mentions additional eligible categories such as Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, eligible federal agencies, regional organizations, U.S. territories or possessions, and non-U.S. (foreign) entities. This wide eligibility reflects an intent to capture diverse cohorts and encourage participation from institutions serving historically underrepresented populations, as well as from international groups that may hold uniquely valuable pediatric datasets.

Administratively, the opportunity is a discretionary NIH grant with a health focus (CFDA 93.310) and uses the X01 mechanism, which is typically associated with providing access to NIH-supported resources or services rather than providing direct research dollars in the way an R01 might. The posting indicates an original closing date of February 19, 2021, and a creation date of October 27, 2020. While the notice does not specify an award ceiling or expected number of awards in the provided text, the practical "award" in this context is the sequencing services and incorporation of resulting data into the Kids First Data Resource, contingent on the cohort being appropriate, the samples being suitable, and the accompanying data meeting program needs.

Overall, this opportunity is best understood as a pathway for investigators who already have access to pediatric cancer or structural birth defect cohorts and can provide high-quality samples plus strong clinical/phenotypic annotation. In exchange, Kids First helps generate comprehensive genomic data (primarily whole genome, with potential exome/transcriptome in justified cases) and then makes those data broadly usable by depositing them into a shared pediatric data resource. The intended impact is faster, more scalable discovery of genetic contributors to childhood cancers and congenital structural anomalies, driven by standardized sequencing and open, community-oriented data reuse.

• 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 2020-10-27.
• Applicants must submit their applications by 2021-02-19. (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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