Innovation Through Collaboration: Academic Annual Report 2016-2017
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Children’s Research Institute Innovation through collaboration Children’s National Health System Academic Annual Report 2016–2017 Vision Children’s National Health System aspires to be a top-five academic pediatric health system that is recognized as leading the quest to prevent or cure many of childhood’s most serious and prevalent disorders. We will achieve this vision through a unique collaboration between clinical and research programs, innovative educational programs, enhanced academic partnerships, improved infrastructure, and a stable base of financial support. Through this approach, our role as a national and international leader in the research and treatment of childhood diseases will be significantly strengthened. Contents From the Directors .............................................................................................................2 CRI Leadership .....................................................................................................................4 Major Scientific Advances 2016 ...................................................................................5 Innovation Spotlight: Gamifying E-Learning to Enhance Medical Education ............................................................................................................. 9 Leading Geneticist and Pediatrician Eric Vilain, MD, PhD, to Lead Center for Genetic Medicine Research at Children’s National Health System .................................................................................................................... 11 Children’s National to Expand Pediatric Research and Collaboration at Former Site of Walter Reed Army Medical Center .......................................12 In Memoriam: Mary Callaghan Rose, PhD............................................................13 Philanthropy: Philanthropic Collaboration Leads to Innovation ...............14 2016 Research Funding .................................................................................................19 Children’s National Intellectual Property Summary ........................................ 20 Clinical and Translational Science Institute at Children’s National (CTSI-CN) .............................................................................................................................21 Center for Cancer and Immunology Research ................................................. 25 Center for Genetic Medicine Research ..................................................................37 Center for Neuroscience Research ..........................................................................55 Sheikh Zayed Institute for Pediatric Surgical Innovation ...............................65 Center for Translational Science ...............................................................................79 Academic Affairs ...............................................................................................................93 Office of Medical Education ........................................................................................97 Selected Grants ...............................................................................................................101 Academic Annual Report 2016–2017 1 From the Directors Our peer-reviewed grant portfolio increased in xcellence is never static. Changes in our organization are expected, and they are embraced for the challenges breadth of funding. This E and opportunities offered. The 2016–17 academic year has been one with several successful transitions in past year we submitted leadership for Children’s Research Institute (CRI). Last year, Eric Hoffman, PhD, our long-term director of CRI’s Center 426 grants (244 federal). for Genetic Medicine Research, departed to become CEO of a thriving start-up company. Children’s National holds an equity stake in the company, and we will continue to benefit from Dr. Hoffman’s leadership. Our comprehensive recruitment search netted an accomplished successor, research enterprise over the next decade. It is also planned to Eric Vilain, MD, PhD, from UCLA. Dr. Vilain will start include incubator and accelerator space for start-ups, industry directing the center in July 2017 (see related article, p.11). partners, and federal and regional academic institutions In addition, Mendel Tuchman, MD, who has served since performing child health research and technology transfer. We 2005 as Chief Research Officer of Children’s National and have chosen a master planner for the site and hope to start scientific director of CRI, announced his plans to retire in occupying the new campus in 2020. July 2017. Fortunately, he has agreed to remain as a senior faculty member for at least one additional year. To succeed Technology transfer has been an area of significant growth. Dr. Tuchman, we recruited Vittorio Gallo, PhD. Dr. Gallo Our faculty has founded 15 start-up companies. This has served for more than a decade as CRI’s Director of the year we completed four licensing agreements for industry Center for Neuroscience Research. We have embarked on to bring therapeutics and devices to commercial use. national searches to replace Dr. Gallo and Yang Liu, PhD, Products in development range from cellular and molecular the Director of the Center for Cancer and Immunology immunotherapy to robotic devices for pediatric surgery and Research. After completing his tenure at the center, Dr. Liu software applications for genetic diagnosis. will collaborate with an industry partner to found and direct a new CRI institute that focuses on molecular immunotherapy. Several benchmarks indicate continued growth of our We thank our outgoing leaders for their tremendous work on research. More faculty and staff are engaged in scholarly behalf of CRI and wish our advancing and incoming leaders activities, as reflected by their greater than 1,000 publications much success in their important new roles. in the medical literature in 2016. The number of principal investigators conducting research projects has grown to The acquisition of land for new facilities brings innumerable 230, and total research annual support reached $73 million. opportunities and the promise of even greater innovation. We have submitted 426 grant applications during the Children’s National procured an almost 12-acre parcel past year. Our investigators have also published seminal of land and several buildings on the Walter Reed Army articles, including a New England Journal of Medicine article Hospital Campus in the District of Columbia. The land, (Driggers) on the Zika virus effect on brain development three miles from the hospital, includes the fabled Armed in the fetus and a Science Translational Medicine article Forces Institute of Pathology research building. The site will (Morton) on the effect of congenital heart disease on be the future home to an innovation district that will include neurogenesis and cortical growth. our research facilities, allowing for a marked expansion of our 2 Children’s National Health System From the Directors Over the past two years, our six signature institutional Under its new director, the Center for Genetic Medicine National Institutes of Health (NIH) grants, valued at more Research will lead a hospital-wide initiative on precision than $40 million, came up for competitive renewal, and medicine. In addition, a number of new faculty members each was successfully re-funded. These awards include the have been recruited to focus on disease mechanisms and Clinical and Translational Science Award (the only one of muscle biology. Strong research on airway diseases continues 64 nationally given to a children’s hospital), the Intellectual to expand, even as we mourn the passing of Mary Callaghan and Developmental Disabilities Research Center, the Rare Rose, a highly accomplished airway investigator and wonderful Diseases Clinical Research Center (received a perfect score human being. A faculty member at CRI for more than 30 of 10), the Child Health Research Career Development, years, she is deeply missed by all who knew her. the Pediatric Emergency Care Applied Research Network (PECARN) Research Network in Emergency Medicine, and The Center for Neuroscience Research competitively the Collaborative Pediatric Critical Care Research Network renewed the NIH-funded District of Columbia Intellectual (CPCCRN) Research Network in Critical Care Medicine. and Developmental Disabilities Research Center (IDDRC). Receiving NIH funding for these and other grants establishes Funded by the NIH since 2001, the IDDRC provides Children’s National as one of the most prominent pediatric a rich environment for performing fully translational academic institutions in the United States and the world. research in the four collaborating DC academic medical centers—Children’s National Health System (lead), The The connection of research to our clinical care programs, George Washington University, Howard University, and one of our best strengths, is only enhanced when research- Georgetown University. intensive clinical division chiefs are in place. We are pleased to note that the six division chiefs recruited in the past The Center for Translational Science; its partner, the year are all NIH-funded investigators, including those in Clinical and Translational Science Institute at Children’s Critical Care Medicine, Nephrology, Emergency Medicine, National; and our affiliated university, The George Hematology, Adolescent Medicine, and Allergy/Immunology. Washington University, competitively renewed the Clinical