Kids First: A New Vision for Pediatric Research

In the world of childhood cancer and congenital disorder research, ten years is more than a milestone—it’s a movement.

Each year in the United States, nearly 16,000 children are diagnosed with cancer before their 20th birthday. Three percent of all children are born with congenital disorders like orofacial clefts or heart defects. Each one of those numbers is really a story of families navigating diagnoses with too few answers, and of researchers determined to change that.

Nine-year-old brain tumor patient Gabriella Miller knew the urgency firsthand. Before she passed, she called on Congress to “stop talking and start doing.” Her advocacy sparked the 2014 Gabriella Miller Kids First Research Act, launching a pediatric research program with a singular mission: know better, do better for children with cancer and congenital disorders.

Today, the Gabriella Miller Kids First Pediatric Research Program (Kids First) stands as a global model for data-driven discovery. In its first decade, Kids First has gone from a visionary idea to pushing the boundaries of pediatric science, precision medicine, and collaborative research.

The Early Years: A Foundation Built from the Ground Up

“When we started, there was no infrastructure—no single place for pediatric researchers to access data across conditions,” recalls Allison Heath, PhD, Director of Data Technology and Innovation for the Kids First Data Resource Center (DRC), who helped write the original grant for Kids First DRC and later joined the team at CHOP. “We were just building something novel. Not just a platform—we were building trust, collaboration, and an entirely new approach.”

That approach was radical at the time: cloud-based from day one, cross-condition by design, and grounded in open science. Researchers from the cancer and congenital disorder fields were brought together in what was once considered an unusual pairing. But Kids First proved that combining data across conditions didn’t dilute impact—it multiplied it.

By 2018, the Kids First DRC launched with the first datasets. Whole-genome sequencing and clinical annotations were made available at no cost to investigators, without embargo (which delays researcher access). “It was a leap forward in how data could be shared, searched, and analyzed,” said Heath. “We were doing things that just hadn’t been done well before.”

But the numbers alone don’t tell the full story. “I remember a webinar where two investigators, totally unplanned, realized they were both seeing the same gene pop up in their research,” said Heath. “That moment of serendipity—that’s what we’re trying to accelerate.”

Kids First DRC’s infrastructure has become so deeply embedded in pediatric research that, as Heath notes, “people just expect us to be there.” And that’s the goal.

Building for the Future, Year by Year

The true success of the Kids First program can be found in the steady, strategic growth of a resource that has become essential to pediatric research. Each step forward has laid the groundwork for the next leap. Here’s a look back at some of the key turning points from the past decade:

• 2015: Program Launched — With Gabriella Miller’s challenge in mind, Kids First was officially funded through the NIH. Behind the scenes, the team began building a completely new infrastructure for pediatric data sharing.
• 2018: Public Portal Launch — The Kids First DRC Portal went live, providing researchers worldwide with free access to whole-genome sequencing data and clinical records. That same year, Kids First hosted its first public webinar and participated in major research conferences, such as the American Society of Human Genetics (ASHG).
• 2019: Tools to Empower Researchers — Features like the Cohort Builder and searchable ontology terms were introduced, giving users more power to find meaningful patterns across datasets. The first peer-reviewed publications using Kids First DRC data were published, marking an early validation of its value.
• 2020–2021: Momentum Builds — As the user base grew, so did demand. By 2021, the number of dbGaP data access requests had passed 100. Imaging data, new disease cohorts, and multi-modal tools were added. “People were motivated—they weren’t just accessing data, they were building discoveries,” recalls Heath.
• 2022–2023: Breaking Silos with Technology — Clinical annotations expanded across thousands of disease-specific terms. AI and machine learning tools were integrated, and CAVATICA became a tool for shared, cloud-based analysis. Kids First’s role in cross-condition research became a model for the field.
• 2024–2025: Modernization and Scale — The second iteration of the Kids First DRC portal launched with an upgraded infrastructure. The RADIANT initiative introduced next-gen AI tools to the platform. The number of genomic files topped 1.1 million, and the program surpassed 1,000 dbGaP requests—fulfilling a vision many doubted was even possible. “I still joke with that investigator,” Heath smiled, “he owes me a beer.”

Through each chapter, Kids First has shown what’s possible when a program refuses to stand still—and when it centers its mission on the children and families who need discovery the most.

A Research Landscape Transformed

Through collaborative cloud platforms like CAVATICA, researchers from institutions large and small can now conduct joint analyses in real time. Imaging data and structured clinical annotations are integrated into multi-modal research, enabling deeper discoveries across cancer subtypes and congenital anomalies.

And now, cross-condition research is no longer the exception. It’s becoming the standard.

“Genomics has moved from research to clinical care,” Heath explains. “Whole genome sequencing is now part of clinical diagnostics. And Kids First is positioned to be the reference point that clinicians use to interpret those findings, so that what’s discovered today can help the next child tomorrow.”

The Road Ahead: Real-Time Discovery, Real-World Impact

The future of the Kids First program is brighter than ever. With the launch of the portal upgrade and AI-powered tools like RADIANT, researchers can explore, filter, and analyze data faster and more intuitively than ever before. New workflows simplify variant discovery, helping scientists move from data to insight more efficiently.

And perhaps most importantly, Kids First DRC ecosystem is closing the gap between research and real-world care. Its datasets are already contributing to more targeted treatments and diagnoses. The next phase of the program will accelerate that translational impact, making discoveries accessible not just to scientists but to clinicians and families.

Join Us in the Next Chapter

The Gabriella Miller Kids First Pediatric Research Program began with a bold challenge. Today, it stands as proof of what’s possible when that challenge is met with innovation, collaboration, and purpose.

We’ve made it easier than ever to get involved.

Researchers: Sign up for access and explore data in the Kids First Portal.

Patient Support Community: Connect with us to support discovery and share your story.

Learn More: Get details about the program and recent funding opportunities.

The next decade holds limitless potential for the Kids First program. Together, we can continue to make groundbreaking discoveries—and bring hope to children and families around the world.