New CDI Studies Underway
The Children’s Discovery Institute recently allocated $2.8 million toward comprehensive genome analysis to improve the ability to diagnosis birth defects, toward work that will seek to identify biomarkers of cognitive dysfunction in survivors of pediatric brain tumors and more.
The CDI also will fund the creation of the Zebrafish Models for Pediatric Research Cooperative (ZRSC), under the direction of Lilianna Solnica-Krezel, PhD. Zebrafish make for great model organisms for genetic dissection of development, regeneration and modeling human birth defects and diseases. These little swimmers can be put through gene disruption, gene editing and trans-genesis, epigenetic profiling and pilot chemical screens for a relatively low cost. The ZRSC will enable new projects that use zebrafish to understand, diagnose and develop treatments for birth defects and pediatric disease.
This funding cycle also launched a study by two School of Medicine leaders in the worldwide malnutrition battle: Indi Trehan, MD, PhD, and Mark Manary, MD, pediatrics. Severe acute malnutrition afflicts 20 million children worldwide at any given time. Through the years, different types of ready-to-use therapeutic foods have helped children with malnutrition but have not addressed the cognitive deficits that often result from it. The CDI researchers will use their funding to test two improved ready-to-use therapeutic food formulas to see which is better at solving for both healthier nutritional status and improved neurocognitive development.
In addition, McDonnell Pediatric Cancer Center researcher, Joshua Rubin, MD, PhD, pediatrics, teams up with Bradley Schlaggar, MD, PhD, pediatrics and neurology, to discover ways to avoid creating cognitive deficits while treating children with brain tumors. Cutting-edge neuroimaging and advanced computational analysis will help the researchers more precisely pinpoint and validate biomarkers for cognitive deficits. This will lay the ground work for more sensitive and truly personalized metrics for improving cognitive outcomes for the ever-growing population of pediatric brain tumor survivors.
In a three-year project, F. Sessions Cole, MD, pediatrics, and a multidisciplinary team will work with investigators and the McDonnell Genome Institute to conduct a comprehensive genome analysis of infants born with birth defects. The goal is to accelerate the state of the art of precision medicine strategies in infants with birth defects; improve diagnostic success; and define genome-directed disease categorization, prognosis, therapeutic strategies and future genetic risk.
“The board’s support of these new projects is advancing the CDI’s vision of applying precision medicine to childhood disease,” says Gary Silverman, MD, PhD, pediatrician-in-chief at Children’s Hospital and CDI executive director. “The CDI will have a major focus in the next 10 years on developing genomics-based personalized treatments for children with common disease, such as asthma and childhood infections, as well as those with rare and neglected disease, birth defects or neurological and neurobehavioral disorders.”