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New hospital center aims at targeted treatment for kids

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Researchers at a new center at the Children’s Hospital of Philadelphia plan to use information generated there to develop targeted disease treatments tailor-made to a child’s genetic profile. The program could also turn out to be a new funding model for research hospitals.

The new Center for Applied Genomics is tasked with pinpointing the genes responsible for asthma, diabetes, ADHD, pediatric cancer, and a host of other diseases that affect young people.

“As a leading children’s hospital, we feel obligated to employ this technology for the benefit of children,” says Philip Johnson, chief scientific officer at CHOP. “We’re all about pediatric healthcare and doing this genomics approach for kids.”

The center will genotype roughly 100,000 children over the next three years. Patients in the hospital’s network, as well as their parents, will be asked to submit blood samples to the study. An advanced information management system will track and triple-encrypt the data, all of which will be made available to the public in an anonymized way.

Heading up the center is Hakon Hakonarson, formerly of Reykjavik-based DeCode Genetics. Hakonarson, who served as director of respiratory, inflammatory, and pharmacogenomics research and then later as vice president of business development, helped lead many of DeCode’s genome-wide mapping and association studies.

The hospital center’s ultimate goals are to design new DNA-based diagnostic tests and identify the relevant biological pathways in order to develop innovative therapies that will tackle the cause, not just the symptoms, of the diseases, says Hakonarson.

The center is using Illumina’s SNP genotyping platform. While Johnson says current SNP technology will suffice for now, down the road the center may consider high-throughput sequencing technology if costs decrease enough to make it feasible. “Could we say that in five years … we’ll just simply put a drop of DNA in a vial and out will pop 3 billion base pairs? Maybe,” he says. “But I think for the short term, our approach of doing the SNP analysis, haplotyping if you will, is probably the technology” that will be the center’s mainstay.

So far, the hospital is funding the center entirely on its own, but it hopes to secure external grants, contracts, and partnerships with pharmaceutical companies further down the road, says Johnson. He also predicts that other hospitals around the nation will soon follow suit with similar self-funded programs.

“I think initially, we anticipate seeing a handful of very large centers who can do the venture capital approach,” says Johnson. “And then down the road, as more targets are identified and the technology becomes more affordable, you’ll see medium-size institutions getting into the business.”

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