In 2014, Congress passed The Gabriella Miller Kids First Pediatric Research Act. The act was intended to be a 10-year program. With the $126 million in funding provided in the original 10 year Act, the National Institutes of Health (NIH) Common Fund established the very successful Gabriella Miller Kids First Pediatric Research Program (Kids First). Since its inception, the program has generated the largest molecular and clinical data sets with approximately 50,000 genetic sequences that are available to researchers and the public. Kids First developed and implemented a transformative infrastructure that the NIH embraced and is using as a template across the Institutes. The program was selected to lead the development of new technologies that will empower the use of electronic health records, in addition to creating a Data Resource Center, and funding 67 grants to research childhood cancer & birth defects.
The Kids First Program fosters collaborative research to uncover the genetic etiology of childhood cancer and structural birth defects. In pursuit of this goal, NIH developed the Gabriella Miller Kids First Pediatric Data Resource Center (Kids First Data Resource Center), and and features a public-facing, web-based portal that allows researchers to search, access, aggregate, analyze, and share annotated genomic sequence, variant, and phenotypic datasets. This resource is highly valued by the pediatric research community and facilitates analyses across diverse conditions to uncover shared developmental pathways. Under the program, more than two-dozen pediatric research focused laboratories have generated vital genomic data that is shared with the entire research community. The overall goal is to help researchers understand the underlying mechanisms of these conditions leading to more refined diagnostic capabilities and ultimately more targeted therapies or interventions. This First Act is just the beginning, we must continue and enhance this vital work, because while cancer is the single leading cause of death by disease among American children, currently only 4% of the National Cancer Institute’s $4.9 billion budget goes towards development of cures and treatments for childhood cancer. Due to limited research in the past 30 years, only 6 drugs have been developed exclusively to treat childhood cancer.
Because of the successes of Kids First Program and its Data Resource Center, Representative Jennifer Wexton (D-VA10), along with 17 other bipartisan cosponsors, introduced the Gabriella Miller Kids First Research Act 2.0 (HR 623) which will reauthorize Kids First and will, over the next five year period, be able to expand a truly comprehensive SHARED-DATA resource for scientists researching the majority of pediatric cancers and rare disease, and will continue to support development of computational tools to analyze very large, complex genomic, and clinical data sets.
Cosponsors include more than 100 Representatives and 7 Senators. On May 18, 2022, Kids First 2.0 was overwhelming passed (53 to 0) by the full House Energy and Commerce Committee and amended to a five-year reauthorization of $25 million per year. As of this writing, there are over 300 foundations and organizations including the Alliance for Childhood Cancer and more than 1,600 advocates covering all 50 states, who have signed a letter of support to the House and Senate for this new bill.
Your support is essential now to build upon the successful work that has already been done and to expand and enhance our capabilities in the future. Please consider cosponsoring H.R. 623, S.1521 – Gabriella Miller Kids First Research Act 2.0
Ellyn Miller, always Gabriella’s mom