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Program Snapshot

Childhood cancers and structural birth defects have profound, lifelong effects on patients and their families. While researchers are increasingly identifying the underlying biological causes of these conditions, the role of genetics is not yet fully understood. A better understanding will spur development of prevention, early detection, and therapeutic interventions. The Common Fund’s Gabriella Miller Kids First Pediatric Research Program (Kids First) will capitalize on exciting advances in genetics research by developing a large-scale data resource. The resource will allow researchers everywhere access to vast amounts of childhood cancer and structural birth defects genetics data that will greatly accelerate their research. In addition, there is considerable scientific evidence that examining childhood cancer and structural birth defects together will uncover new connections between them that would not have been uncovered had one or the other been examined independently. By integrating large amounts of genetic data from patients with childhood cancer and structural birth defects, Kids First will facilitate new discoveries and novel ways of thinking about these conditions. This is anticipated to accelerate scientific progress in pediatric research that will improve the lives of the children and families impacted by these conditions.

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Program Highlights

Kids First is excited to announce the selection of a second set of childhood cancer and structural birth defects patient cohorts for whole genome sequencing. These patient cohorts will contribute to the forthcoming Kids First Data Resource which will allow scientists to identify genetic pathways that underlie these conditions and to explore whether shared genetic pathways exist between childhood cancer and structural birth defects. The applications were reviewed by a panel of external scientific experts and the selected cohorts address the following conditions:

  • Cancers of the nerves that help control involuntary function (Neuroblastoma)
  • Cancers of the bone marrow that makes blood cells (Acute lymphoblastic leukemia and Hodgkin and non-Hodgkin lymphoma)
  • Diverse collection of Central Nervous System (CNS) and non-CNS solid tumors
  • Congenital Heart Defects
  • Developmental disorders of the chest muscle used for breathing (Congenital Diaphragmatic Hernia)
  • Cleft lip and cleft palate (Orofacial Cleft Birth Defects)
  • Congenital hearing loss
  • Abnormal curvature of the spine (adolescent idiopathic scoliosis)

Learn more about the 2015 and 2016 cohorts. The Kids First program plans to call for applications to support whole genome sequencing of additional childhood cancer and structural birth defect cohorts in fiscal years 2017-2020, pending availability of funds.

Updates from the Kids First Program

"Less talk, more action," was a common refrain for 10-year-old Gabriella Miller
The Gabriella Miller Kids First Research Program was featured in a Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) spotlight. The spotlight talks about how the program began, how it is managed, and why it is focused on studying childhood cancer and structural birth defects together.

Kids First Community Webinar
The Kids First program hosted a community webinar on November 18th to discuss the current progress of the Kids First program and its plans for the future. A recording of the webinar and a pdf version of the slides are available. Sign up for the Kids First email listserv to receive program updates and to ask questions or provide comments concerning the Kids First program. We look forward to hearing from you.

Genetic sequence and phenotype data from Kids First cohorts will be publicly available beginning February 2017
In FY2015, Kids First selected seven cohorts of children with cancer or structural birth defects for whole genome sequencing. The DNA sequence data along with well-curated clinical phenotype data will be deposited into the forthcoming Kids First Data Resource. Researchers everywhere will be able to use the data to identify genetic pathways that underlie these conditions and to explore whether shared genetic pathways exist between childhood cancer and structural birth defects. Until the Kids First Data Resource is completed researchers can visit the research projects page for the data release dates for each of the cohorts and the FAQs page for guidance on how to access the data.

New Funding Opportunity to analyze childhood cancer and/or structural birth defects genomics datasets
NIH is soliciting applications to analyze childhood cancer and/or structural birth defects data that will be part of the Kids First Data Resource or could be included in the Kids First Data Resource. Development of statistical methodology appropriate for analyzing genome-wide data may also be proposed. Standard application due dates apply.

 
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