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Welcome to Discovery to Innovation

Discovery to Innovation is a quarterly update of news, research findings, and game-changing developments at
The Childrenʼs Hospital of Philadelphia Research Institute.

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The Children's Hospital of Philadelphia Research Institute was recently the site of a meeting of more than 350 genomics researchers, pharmaceutical executives, and policymakers. Held in the Colket Translation Research Building, the ICG Americas 2012 conference was the first international meeting on genomics to be held in the United States, and was hosted jointly by international genomics institute BGI and Children's Hospital.

Among the more than 50 speakers at the ICG Americas 2012 conference were CHOP's own Hakon Hakonarson, MD, PhD, director of the Center for Applied Genomics (CAG), and Douglas Wallace, PhD, director of the Center of Mitochondrial and Epigenomic Medicine. Eric Green, MD, PhD, director the National Human Genome Research Institute (NHGRI), and Huanming Wang, PhD, chairman of BGI, also presented.

Dr. Hakonarson's talk highlighted his work with a fasoracetam, a drug that was originally developed to treat Alzheimer's disease but, after being put through clinical trials, was shelved for efficacy reasons. Dr. Hakonarson and his team are currently investigating whether fasoracetam could be used to treat ADHD, a project he called a "representation of what genomics is offering."

Because mutations of the metabotropic glutamate receptor (mGluR) are found in 15 to 20 percent of ADHD patients, Dr. Hakonarson and his team are studying whether fasoracetam — an mGluR agonist — could be an effective treatment for ADHD in that patient population. That fasoracetam has already been through a battery of trials — 28 in all — allows for a truncated drug development timeline, and using genomics to determine new indications for available products can help speed therapies to market.

Genomic medicine allows researchers "to identify a subset of patients who have mutations in certain gene pathways and networks that disturb the networks and, when you are lucky and a drug already exists that has already been proven safe, the ability to take a shortcut and fast-track a clinical trial and subsequently get a drug out is obviously significantly enriched," Dr. Hakonarson said.

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