Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects: Gabriella Miller Kids First Pediatric Research Program (X01) | Apply Online

FAQs: Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects (Kids First X01) - PAR 16-150

What is this NIH funding opportunity?

This is an NIH opportunity titled "Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects: Gabriella Miller Kids First Pediatric Research Program (X01)" (Funding Opportunity Number PAR 16-150). It supports research that uses whole genome sequencing (WGS) to uncover how genetics contributes to childhood cancers and structural birth defects.

What is the main goal of the program?

The goal is to generate high-quality whole genome sequencing data from well-characterized pediatric cohorts and use those data to identify genetic variants and genomic patterns that may explain disease risk, disease mechanisms, and relevant biological pathways. A major objective is also to grow a shared data resource that benefits the broader pediatric research community.

What diseases or conditions does the opportunity focus on?

The scientific focus is on two major pediatric disease areas: childhood cancers and structural birth defects.

What type of data does this program emphasize generating?

The opportunity emphasizes generating whole genome sequencing (WGS) datasets. WGS is intended to provide a comprehensive view of the genome, beyond what smaller gene panels or exome sequencing might capture.

Does this opportunity pay for applicants to run sequencing in their own labs?

A defining feature of this X01 mechanism is that it provides access to whole genome sequencing performed at a Kids First-supported sequencing center, rather than primarily providing a traditional research budget for applicants to run sequencing independently.

Where is the sequencing performed?

Sequencing is conducted through designated program sequencing centers supported by the Kids First program. This approach is intended to ensure consistent methods, quality control, and harmonized outputs across projects.

What do applicants propose under this opportunity?

Applicants propose pediatric cohorts and the research questions to be addressed. The program-supported sequencing centers then perform the whole genome sequencing for the selected projects.

What happens to the genomic data generated through this program?

The resulting genomic datasets are deposited into the Gabriella Miller Kids First Pediatric Data Resource, a shared data platform designed to make these datasets available to the wider pediatric research community.

Is data sharing required?

Yes. The program explicitly expects that generated datasets will not remain only within the applicant team and will be contributed to the Kids First Pediatric Data Resource to support broad community use.

How does this opportunity benefit the broader research community?

Each funded project is expected to both advance a specific scientific question and add durable community value by expanding the size, diversity, and utility of the Kids First data ecosystem through shared, standardized WGS data.

What kinds of genetic discoveries are encouraged for childhood cancer studies?

The announcement highlights identifying inherited (germline) variants that may increase predisposition, characterizing tumor genomes when relevant, and examining broader genomic architecture such as structural variants, copy number changes, and noncoding variation that may be missed by exome sequencing or smaller panels.

What kinds of genetic discoveries are encouraged for structural birth defect studies?

The opportunity targets genetic etiology of structural birth defects, where WGS can help detect de novo mutations, rare inherited variants, and larger-scale genomic rearrangements that may underlie congenital anomalies.

Why does the program emphasize whole genome sequencing instead of exome sequencing or panels?

Because pediatric cancers and structural birth defects can involve complex genetic mechanisms, WGS is emphasized to capture a comprehensive view of the genome, including noncoding variation and larger structural changes that may be missed when focusing only on protein-coding regions.

Who is eligible to apply?

Eligibility is broad and includes many domestic organization types commonly eligible for NIH opportunities. The listed eligible applicants include state, county, and city or township governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments and other tribal organizations; public housing authorities/Indian housing authorities; nonprofit organizations with or without 501(c)(3) status (other than higher education institutions); for-profit organizations other than small businesses; small businesses; and additional entities as described in the full eligibility text.

Why is eligibility so broad?

The program aims to draw from a wide range of organizations that can assemble appropriate pediatric cohorts, including clinical networks, registries, biobanks, research consortia, and institutions with strong phenotypic data and consent suitable for genomic analysis and data sharing.

What is the award type and health category listed for this opportunity?

The award is categorized as a discretionary HHS/NIH grant in the health activity category, with CFDA 93.310.

What is the Funding Opportunity Number?

The Funding Opportunity Number is PAR 16-150.

What is the mechanism mentioned in the title?

The mechanism referenced in the title is X01, described in the provided information as a gateway to generate standardized, program-supported whole genome sequencing data paired with an expectation of data contribution to a shared national resource.

When was this opportunity posted and when did it close?

The opportunity was posted and created on March 24, 2016. The original and current closing date listed is June 17, 2016.

Does the listing provide an award ceiling or expected number of awards?

The provided fields do not specify an award ceiling or the expected number of awards. This is described as consistent with some NIH announcements where the primary resource being provided is sequencing capacity and associated program support rather than a simple dollar cap in a summary table.

Which agency issued the opportunity?

The agency is listed as HHS-NIH11, indicating an NIH issuing component under the Department of Health and Human Services (HHS).

What is the Gabriella Miller Kids First Pediatric Research Program?

It is a broader NIH-associated program created to accelerate discovery in pediatric cancer and structural birth defects through large-scale genomic data generation and broad data sharing.

What is the Kids First Pediatric Data Resource?

It is the shared data platform where resulting genomic datasets from this program are deposited so they can be made available to the wider pediatric research community.

What is meant by "well-characterized pediatric cohorts" in this context?

Based on the provided description, the program emphasizes cohorts with strong characterization to support genetic discovery, including well-defined participant groups and strong phenotypic data, along with consent suitable for genomic analysis and data sharing.