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NIH selects OHSU Knight Cancer Institute to co-lead largest national database for pediatric cancer, rare disease research

The “Kids First” grant caps off a momentous time, with three prestigious national computational biology grant awards totaling $17 million in funding to OHSU
Adam Margolin, Ph.D.
Adam Margolin, Ph.D., professor of biomedical engineering, and director of computational biology in the OHSU School of Medicine and the OHSU Knight Cancer Institute. (OHSU/Kristyna Wentz-Graff)

The National Institutes of Health announced today that it is establishing a pediatric cancer and rare disease data program that will allow clinicians and researchers to access multiple datasets in one location -- a first for the pediatric research community.

The NIH Health Common Fund's Gabriella Miller Kids First Pediatric Research Program is awarding a $15 million grant to establish the Kids First Data Resource Center. The five-year award, contingent on available funding, will allow scientists to harness genomic data from thousands of pediatric patients to collaboratively engage in shared research on behalf of children diagnosed with cancer and other diseases.

"There is an unprecedented amount of genomic data available, and these data hold the promise of yielding breakthroughs for debilitating pediatric diseases. The idea behind the DRC is to unlock this potential by allowing researchers and clinicians across the world to easily apply advanced analytics to as much pediatric cancer and rare disease data as possible,” said Adam Margolin, Ph.D., co-principal investigator on the project, professor of biomedical engineering, and director of computational biology in the OHSU School of Medicine and the OHSU Knight Cancer Institute.

OHSU will collaborate with investigators from the Center for Data Driven Discovery in Biomedicine (D3b) at the Children's Hospital of Philadelphia, biomedical data analysis firm Seven Bridges, the Ontario Institute for Cancer Research, the University of Chicago, and Children's National Health System.

The Data Resource Center will provide new analytic tools and access to the largest cross-disease collection of annotated clinical and genomic sequence data from children with cancer and structural birth defect and their families. At the inception of the project, the team will immediately work to make available genomic sequencing data, and associated tools, from 6,000 pediatric patient samples.

By enabling researchers to find and perform large-scale analyses across pediatric cohorts, the DRC will act as a centralized hub from which researchers and clinicians can access phenotypic and genetic sequence data for novel research. Other shared data resources exist in pockets across the country, but the DRC is the largest culling of that information in one resource, according to Margolin.

"NICHD is committed to supporting research on birth defects as part of its overall focus on improving the health of children,” said Diana Bianchi, M.D., NICHD director. "The Kids First initiative provides a unique opportunity to use DNA sequencing information to gain a better understanding of the underlying causes of birth defects and childhood cancers.”

Momentum for OHSU's computational biology program
Being tapped to co-lead an endeavor of this magnitude highlights a momentous few months for the OHSU computational biology program, and continues to fulfill a promise made to donors during the $1 billion fundraising challenge.

"One initiative we promised was the strengthening of computational biology,” said Brian Druker, M.D., director of the Knight Cancer Institute. "Adam has recruited seven faculty, a total of 50 people, and has brought in more than $20 million in grants funding to build a computational program that is bringing nationwide recognition to OHSU. We recognized that this resource would be a necessary part of our overall effort to end cancer as we know it, and we are extremely pleased to see this remarkable return on our investment.”

Over the last three years, the computational biology program has been awarded more than 30 grants, with computational biology personnel as co-principal investigators, totaling more than $22 million to computational biology, $41 million to OHSU, and $90 million for multi-institutional grants.

In June and July, OHSU was selected to join three prestigious national consortia for computational cancer biology research. Margolin will serve as the computational lead and co-principal investigator on the Kids First Award as well as:

  • A $9.2 million National Cancer Institute grant to study triple-negative breast cancer as a Research Center in the NCI's Cancer Systems Biology Consortium. Co-investigators include: Joe Gray, Ph.D., Rosalie Sears, Ph.D., and Claire Tomlin, Ph.D.
  • A $5.8 million NCI grant to study drug response and resistance in acute myeloid and chronic lymphocytic leukemias as part of the NCI's Cancer Target Discovery and Development Network. Co-investigators include Druker and Jeff Tyner, Ph.D.

This year, OHSU also was awarded a $2.1 million grant to serve as the center for RNA sequencing and pathway-based analysis within the NCI's Genome Data Analysis Network. Investigators are Margolin and Paul Spellman, Ph.D.

In addition, the OHSU Knight Cancer Institute recently was awarded the National Cancer Institute's highest distinction as a Comprehensive Cancer Center. By joining four prominent national computational consortia, the Knight Cancer Institute should be well positioned to include leadership in computational biology within its slate of comprehensive clinical and research activities to benefit cancer patients.

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