Abstract
Through a large-scale tumor-sequencing effort for pediatric brain cancer, researchers hope to bring personalized care to young patients.
Researchers recently kicked off an ambitious effort to molecularly characterize a large number of tumor specimens from the Children's Brain Tumor Tissue Consortium (CBTTC; http://cbttc.org). Over the next 6 months, they aim to create the Pediatric Brain Tumor Atlas, which will be the most extensive database of its kind.
Although brain tumors are the leading cause of disease-related death in children, efforts to put big data to work against childhood brain cancer have lagged, partly because, compared with adult cancers, the disease is rare. With only about 4,000 new cases per year in the United States, amassing sufficient samples in any one cancer center has been impossible.
To get around that problem, Adam Resnick, PhD, and colleagues established the CBTTC at Children's Hospital of Philadelphia, PA. With 13 member hospitals around the country, this biorepository currently holds tissue specimens from more than 2,000 cases representing more than 30 types of pediatric brain tumors, all annotated with extensive clinical and phenotypic data.
The tumor atlas will be the first project supported by the pediatric arm of Cancer MoonShot 2020, an industry–academic–philanthropic partnership led by physician-scientist Patrick Soon-Shiong, MD, and independent of the federal government's National Cancer Moonshot. Launched in January 2016, Cancer MoonShot 2020 is focused on accelerating clinical development of cancer immunotherapies.
The CBTTC's tumor specimens are being analyzed at Culver City, CA–based NantHealth via GPS Cancer, a profiling tool that generates whole-genome, RNA-sequencing, and proteomic information. The nonprofit Chan Soon-Shiong Institute of Molecular Medicine in Windber, PA, is funding this effort through a $20 million award.
To date, about one fifth of the samples have been analyzed, and the data are already appearing on CAVATICA, an open-access cloud-based platform recently launched by Resnick and colleagues (available at www.cavatica.org). The rest should be available over the next few months, he says.
“We believe these data should be used for discovery as soon as possible,” he adds. “So, as the samples are analyzed, we are putting the results out immediately, where they can be accessed prepublication with no embargo.”
The atlas's inclusion of extensive clinical data is a unique and useful feature, says the CBTTC's executive chair Rishi Lulla, MD, a pediatric oncologist at the Children's Hospital of Chicago, IL. “In the past, lack of easy access to pertinent clinical information has been a limitation of these large genomic sequencing efforts,” Lulla says.
The atlas also fills a big gap in the effort to bring precision medicine to children with brain cancer, says Sabine Mueller, MD, PhD, a pediatric neuro-oncologist at the University of California, San Francisco. A lot of resources have been devoted to the molecular characterization of adult tumors, but the results have not always translated to young patients.
“Kids are not just little adults; their brain tumors are quite different at the molecular level,” Mueller says. “This data is going to help us to develop specific therapies based on better understanding the unique biology of pediatric brain cancer.” –Pat McCaffrey
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