The National Institutes of Health (NIH) is spearheading a $14.8 million, five-year effort to launch a data resource center for cancer researchers around the world in order to accelerate the discovery of novel treatments for childhood tumors.
Contingent on available funds, five years of funding will be provided by the NIH Common Fund Gabriella Miller Kids First Pediatric Research Program, named after Gabriella Miller, a 10-year-old child treated at Children's National.
As principal investigators, researchers at Children's Hospital of Philadelphia will lead the joint effort to build out the "Kids First" Data Resource Center. Meanwhile, Children's National Health System in Washington, D.C., will spearhead specific projects, including the Open DIPG (diffuse intrinsic pontine glioma, aggressive pediatric brain tumors that defy treatment and are almost always fatal) project, and as project ambassador will cultivate additional partnerships with public and private foundations and related research consortia to expand a growing trove of data about pediatric cancers and birth defects.
The Kids First data resource center is a centralized platform of well-curated clinical and genetic sequence data from dozens of childhood cancer and structural birth defect cohorts, comprising genetic data from thousands of patients and their families. The team will integrate large, disparate data sources, provide support for analyses, and coordinate with third-party data commons and applications, according to a press release announcement.
Researchers can use this resource to probe genetic pathways and explore genetic abnormalities that underlie childhood cancer and structural birth defects. The program will also provide funds to generate new data and facilitate deposition into a centralized database.
Officials noted that it’s important to study these conditions together because children with birth defects are at a higher risk of also developing childhood cancer, suggesting they may share an underlying cause. However, not much is known about these suspected pathways. Few large-scale genetics studies have focused on both childhood cancer and structural birth defects. These shared biological pathways may not be detected if researchers study cancer patients or those with structural birth defects independently.
Two other crucial components of the Kids First project are the teams led by Robert L. Grossman, Ph.D., and Sam Volchenboum, M.D., Ph.D., at the University of Chicago. Grossman and Volchenboum will play a key role in the technical underpinnings of the large-scale processing and sharing of genomic and clinical data for this initiative.
Grossman, the Frederick H. Rawson Professor in Medicine and Computer Science and director of the Center for Data Intensive Science at the University of Chicago, heads up an operations center that runs numerous data commons, supporting more than 20,000 researchers across the world every month. “Platforms that enable researchers to analyze securely large amounts of de-identified clinical and genomic data are one of our most powerful tools for making discoveries that improve children’s lives,” Grossman said in a statement.
The Chicago team of engineers and scientists will design and operate the cloud-based, open-source software needed to establish the data coordination center within the Kids First data resource center.
The NIH grant builds on previous funding that Congress provided to the NIH Common Fund to underwrite research into structural birth defects and pediatric cancers. In the first phase, so-called X01 grantees—including Eric Vilain, M.D., Ph.D., newly named director of the Center for Genetic Medicine Research at Children's National—received funding to sequence genetic data from thousands of patients and families affected by childhood cancer and structural birth defects.
"It's a question of numbers,” Vilain said in a statement. "The bottom line is that making sense of the genomic information is significantly increased by working through large consortia because they provide access to many more patients with the disease. What is complicated about genetics is we all have genetic variations. The challenge we face is teasing apart regular genetic variations from those genetic variations that actually cause childhood cancers, including DIPG."
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