Children’s Research Institute | Academic Annual Report 2017-2018

Innovation Through Collaboration

Letter

From the Directors

Excellence is never static. Changes in our organization are expected and they are embraced for the challenges and opportunities offered.

The 2017-18 academic year has been one with successful transitions in four of the seven leadership positions at Children’s Research Institute (CRI):

  • Eric Vilain, M.D., Ph.D., a pediatric geneticist from UCLA, succeeded Eric Hoffman who left to become CEO of a Children’s National start-up company after 18 years at the helm of the Center for Genetic Medicine Research. Dr. Vilain, whose research focuses on disorders of sexual differentiation, is leading our effort in genomic and precision medicine.
  • Vittorio Gallo, Ph.D., who served 16 years as director of the Center for Neuroscience Research was elevated to chief research officer, succeeding the retiring Mendel Tuchman, M.D., who had held the post for more than a decade.
  • Cath Bollard, M.D., MBChB, who joined Children’s from Baylor College of Medicine, to direct our cellular immunotherapy program succeeds Yang Liu, Ph.D., as director of the Center for Cancer and Immunology Research. Dr. Liu moved to the University of Maryland after five years at Children’s.
  • Anthony Sandler, M.D., surgeon-in-chief, will now direct the Sheikh Zayed Institute for Pediatric Surgical Innovation, succeeding Peter Kim, M.D., Ph.D., who was the founding director.

We are also moving toward physical change at CRI with the acquisition of almost 12 acres of land on the historic Walter Reed Army Hospital site, three miles from Children’s National’s main campus to create a research and innovation campus. The first phase of development is scheduled to open in 2020 and is planned to house our Genomics and Precision Medicine initiative.

Technology transfer also has been an area of significant growth. Our faculty has founded 17 start-up companies. This year, we completed four licensing agreements for industry to bring therapeutics and devices to commercial use. Products in development range from cellular and molecular immunotherapy to robotic devices for pediatric surgery and software applications for genetic diagnosis.

Several benchmarks indicate continued growth of our research. More faculty and staff are engaged in scholarly activities, as reflected by their more than 1,000 publications in the medical literature in 2017. The number of principal investigators conducting research projects has grown to 230, and total research annual support reached $73 million. In addition, we have submitted 426 grant applications in the past year.

The connection of research to our clinical care programs, one of our strengths, is only enhanced by our research-intensive clinical division chiefs. We are pleased to note that the six division chiefs recruited in the past two years are all also funded investigators, including critical care medicine (Michael Bell, M.D., University of Pittsburgh), nephrology (Marva Moxie Mimms, M.D., NIH), emergency medicine (Robert Freishtat, M.D., Children’s National), hematology (Suvankar Majumdar, M.D., University of Mississippi), adolescent medicine (Lisa Tuchman, M.D., Children’s National), and endocrinology (Andrew Dauber, M.D., Cincinnati Children’s Hospital). We also have established a first-of-its-kind Rare Diseases Institute combining clinical care, training, and research, and directed by Marshall Summar, M.D., who also serves as chief of the Division of Genetics and Metabolism, and who led our acquisition of the Walter Reed property.

In support of our faculty, the Clinical and Translational Science Institute at Children’s National Grant Enhancement Program (GEP) continues to expand, assisting an increasing number of investigators with their initial writing and submitting of federal grant applications. Since its inception in 2010, the GEP has received 336 grant applications for review at various stages of development (80 in 2017 alone). Of the 210 proposals subsequently submitted for funding that have been reviewed, 82 (39 percent) were funded. That success has led us to add supported time for more experienced senior faculty to participate in the program. Now, all junior faculty members are required to seek GEP review and assistance for every external grant proposal.

The Center for Cancer and Immunology Research strengthened the cellular and molecular immunotherapy program, which was awarded a $1.5 million grant from the board of visitors as well as a prestigious translational research program grant from the Leukemia Lymphoma Society. Cancer center faculty reported their breakthrough studies in high impact journals, including Cancer Cell, Immunity Cell and the Journal of Clinical Oncology. The center has also developed major collaborations with Seattle Children’s Research Institute (SCRI) and the George Washington University (GW). The collaboration with SCRI is the development of an innovative consortium, Cureworks, to drive immune cell-based therapies for pediatric patients with cancer across the U.S. and the world. The GW collaboration established a memorandum of understanding with the GW Cancer Center, with the intention of applying for designation as a National Cancer Institute designated Cancer Center within 5 years.

Under its new director, the Center for Genetic Medicine Research will lead a hospital-wide initiative on precision medicine, building resources for state-of-the-art genome-wide analysis of diseases, bioinformatics tools to understand the massive amount of genetic information, and pre-clinical models to investigate disease mechanisms and treatments. New faculty members from the fields of muscle biology, reproductive health and gene editing have been recruited to focus on many aspects of genomics and precision medicine. The center started a new bioinformatics initiative to support current and future programs at CRI. This initiative will coalesce knowledge and provide a central location for informaticians who can organize and share expertise, as well as identify gaps and strategic opportunities.

The Center for Neuroscience Research was awarded a number of new NIH RO1 grants and published multiple papers in high profile journals including Nature Communications, Science Translational Medicine, Neuron and eLife. Newly funded RO1s cover diverse basic and translational relevant areas of neurodevelopment and neurodevelopmental disorders such as perinatal brain injury, neuroprotection, fetal alcohol syndrome and genetics of brain development.

The Center for Translational Science (CTS) continues to be organized into three major sub-themes that reflect the broad base of its investigator-initiated research: 1) molecular pathogenesis and experimental therapeutics; 2) patient-oriented research; and 3) behavioral and community research. These sub-themes include investigator-initiated programs, as well as NIH-funded consortia in which Children’s National researchers play leadership roles. Studies conducted by CTS faculty extend along the full spectrum of translational research. The center continues to support a portfolio of special interest groups that serve as “catalyst programs” for specific research themes involving a broad range of investigators within the center and from the greater CRI community.

The Sheikh Zayed Institute for Pediatric Surgical Innovation continues to build upon the collective strengths of its members and collaborators to enable the development of pediatric-labeled medical devices. The institute provides a platform of integrated business, regulatory and scientific expertise to its faculty and other qualified device innovators. In the past year, the institute increased its success rate of federally funded awards and particularly augmented its portfolio of Small Business Innovation Research (SBIR)and Small Business Technology Transfer (STTR) funded projects. In 2017, for the first time, the institute co-located its Symposium in Pediatric Device Innovation with The MedTech Conference, powered by AdvaMed, in San Jose, California. Life-to-date, the institute has guided 79 pediatric medical devices through every stage of the total product life cycle. Of these, 26 devices are internal, and 53 are being developed by external companies.

Our Education and Training Programs continue to excel as well. Children’s National Medical Center is recognized by the Accreditation Council for Graduate Medical Education (ACGME) as a model for educational innovation, demonstrated by our selection as the only pediatric institution to serve as a member of the prestigious Pursuing Excellence Innovator Collaborative. The goal of the ACGME funded grant program is to transform graduate medical education through interprofessional collaboration to improve patient care quality and safety. The robust Children’s National eLearning and Simulation programs are fundamental to achieving this goal. We also promote academic career growth and productivity through our interprofessional faculty academies to support educational and improvement science scholarship. We currently train over 500 medical students and 1,000 residents and fellows each year in 45 specialty programs. Our residency program remains one of the most competitive in the nation, with submissions from two-thirds of all fourth-year U.S. medical students applying for internships in pediatrics. In July 2017, 40 new pediatric interns, selected from more than 2,600 applicants, matriculated at Children’s National. More than 20 percent of the intern class come from underrepresented minority populations.

Finally, a number of our faculty have received recognition nationally. Chief among the honorees was Robin Steinhorn, M.D., president-elect of the Academic Pediatric Society and Anna Penn, M.D., Ph.D., elected as membership director of the Society for Pediatric Research.

In sum, this has been a remarkable year of transitions, continued growth, and distinction at Children’s Research Institute. As always, our achievements, large and small, make profound differences in the lives of the children and families we serve.

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