Gabriella Miller Kids First Pediatric Research Program

Overview

Gabriella Miller Kids First logoThe Gabriella Miller Kids First Pediatric Research Program (Kids First) is an NIH-wide program supported through the NIH Common Fund that was initiated in response to the 2014 Gabriella Miller Kids First Research Act. Kids First fosters collaborative research to uncover the causes of childhood cancers and structural congenital anomalies and support data sharing within the pediatric research community.

In 2018, the program launched the Kids First Data Resource Portal external link through the Gabriella Miller Kids First Data Resource Center external link. The web-based portal serves as the primary entry port to large amounts of genetic and associated clinical and phenotypic data from patients with congenital anomalies and childhood cancers and their families, as well as cloud-based workspace to empower analyses of these data. Both childhood cancers and congenital anomalies are critical and costly conditions associated with substantial morbidity and mortality. Exposing the underlying genetic causes of these diseases has the potential to profoundly improve preventative measures, diagnostics, and therapeutic interventions.

Kids First also collaborates with other NIH programs, institutes, and centers, such as the INvestigation of Co-occurring conditions across Lifespan to Understand Down syndromE project and the National Institute on Alcohol Abuse and Alcoholism, to increase sequencing of Down syndrome and fetal alcohol spectrum disorders cohorts. The program is also working with the Common Fund’s Knockout Mouse Phenotyping Project to validate Kids First findings using mouse models.

Topic Areas

To date, Kids First has selected 63 patient cohorts for whole genome sequencing, which represents approximately 55,000 genomes and 21,000 patients. The datasets collected in the Kids First Data Resource Portal represent more than 30 conditions under active study by investigators, with more likely to be included as the program continues.

Conditions range from types of cancer, such as childhood acute myeloid leukemia and Ewing sarcoma, and cancer susceptibility to congenital structural conditions, such as diaphragmatic hernia and orofacial clefts. The work also addresses less common conditions, such as Cornelia de Lange syndrome and pediatric intracranial germ cell tumors, as well as conditions that co-occur with congenital anomalies, such as Down syndrome. Visit the Data Research Portal external link for more information.

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