Johns Hopkins All Children’s Hospital brings together researchers for the 10th annual symposium to discuss approaches to pediatric research.

Researchers from Johns Hopkins All Children’s Hospital, Johns Hopkins University School of Medicine and other leading academic medical centers shared key findings, strategies and insights at the 10th Annual Johns Hopkins All Children’s Research Symposium, held Oct. 29 in St. Petersburg, Florida, and online.

Hopkins faculty joined invited speakers to share their perspective on different approaches to basic, clinical and translational pediatric research, from randomized clinical trials to consortia focused on very rare diseases to the role of powerful databases for both prospective and retrospective research.

“It is remarkable to see the progress we have made in one decade,” says Johns Hopkins All Children’s Hospital President Alicia Schulhof as she welcomed the presenters and participants. “Our 10th annual symposium beautifully highlights the innovation that will help us continue to improve children’s health through pediatric research.”

Three research leaders at Johns Hopkins All Children’s served as moderators: Ernest Amankwah, Ph.D., director of the Data Coordinating Center for Pediatric Multicenter Studies and Raquel Hernandez, M.D., M.P.H., director of the Center for Pediatric Health Equity Research, both within the Johns Hopkins All Children’s Institute for Clinical and Translational Research; and Timothy Osborne, Ph.D., associate dean for basic research and director of the Johns Hopkins All Children’s Institute for Fundamental Biomedical Research.

The keynote presentation — "A Tale of Two Cities” — highlighted experiences in designing and executing Hopkins-led multicenter trials that inform future clinical care. Daniel Hanley, M.D., professor of neurology, neurosurgery, and anesthesia and critical care medicine at Johns Hopkins School of Medicine in Baltimore, described the importance of bench research in planning clinical trials and the role of education and support in mobilizing participating sites for a multicenter trial.

Neil Goldenberg, M.D., Ph.D., associate dean for research, director of the Johns Hopkins All Children’s Institute for Clinical and Translational Research, and the Perry Family Professor in Clinical and Translational Research, echoed the importance of research infrastructure at participating sites as well as the coordinating center. “In 2021, this requires expertise in design, execution, oversight, data analysis and biospecimen banking.”

A health crisis like COVID-19 can accelerate the launch of a multicenter trial, Goldenberg noted. By June 2020, a team at the Johns Hopkins All Children’s Institute for Clinical and Translational Research had successfully launched a national clinical trial to study the dosing and safety of anti-clotting medications for the prevention of clotting complications in children with COVID-19 illness and the COVID-related multisystem inflammatory syndrome in children (MIS-C).

Other invited lecturers highlighted research needs and trends:

• Harry (Hal) Dietz, M.D., Victor A. McKusick Professor of Medicine and Genetics at Johns Hopkins University and a pioneer in identifying the cause and a treatment for Marfan syndrome, described the importance of translational research in developing precision medicine approaches to genetically based vascular disease.
• Chester Wayne Brown, M.D., Ph.D., professor and division chief of genetics at University of Tennessee Health Sciences Center, stressed the importance of engaging underrepresented populations to increase equity in genomic research on rare diseases.
• Damon Reed, M.D., a sarcoma specialist at Johns Hopkins All Children’s and Moffitt Cancer Center, described the challenges of studying ultrarare cancers and the role of research collaboratives in developing a nimble approach to potential treatments.
• John Cleveland, Ph.D., center director and executive vice president at Moffitt Cancer Center, discussed data as a major driver of research and the role of fostering interaction between clinicians and other scientists.
• Michael Cohen-Wolkowiez, M.D., Ph.D., distinguished professor of pediatrics at Duke University, highlighted the role of computational pharmacology in finding the right dosage for children and in modeling development of new medications.
• Thomas Inge, M.D., Ph.D., professor of surgery and pediatrics at Children’s Hospital Colorado, described the role of surgical consortia and factors for success in multicenter research.

Post-graduate trainees, physicians and faculty based at Johns Hopkins All Children’s and affiliate institutions highlighted their research endeavors in a poster session that preceded the symposium, with several selected for oral presentation and discussion at the full symposium. Congratulations to the authors of the winning abstracts, selected from a total of 48:

• Best Abstract—Trainee Initiated Clinical and Translational Research: Stress Ulcer Prophylaxis for Critical Asthma: A Multicenter, Retrospective Study. Alexa Roberts, M.D., Meghan Roddy, Pharm.D., Michael Wilsey, M.D., Scott McKinley, D.O., and Anthony Sochet, M.D.
• Best Abstract—Clinical and Translational Research, Faculty-Initiated: Suicide, Stimulants and SSRIs: A Retrospective Chart Review. Reena Thomas, M.D., Austin Sellers, B.S., Jamie Fierstein, Ph.D., Mark Cavitt, M.D., and Jeffrey Alvaro, M.D.
• Best Abstract—Basic Science, Trainee-Initiated: Akt3 Activation by R-Ras Stabilizes Endothelium via Intercellular Crosstalk Mediated by Jagged 1-Notch. Jose L. Herrera, Ph.D., Masanobu Komatsu, Ph.D.
• Best Abstract—Basic Science, Faculty-Initiated: Multiorgan Human-On-A-Chip Model of the Gut-Liver-Brain Axis Reveals New Insights into Metabolism and Autoimmunity. Martin Trapecar, Ph.D.

“Both the quantity and quality of research studies on our campus continue to grow,” notes George Jallo, M.D., vice dean and physician-in-chief at Johns Hopkins All Children's Hospital, David M. Goldenberg Family Chair in the Institute for Brain Protection Sciences, and a professor of neurosurgery, pediatrics and oncology with Johns Hopkins University School of Medicine.

“Our resident physicians, clinical and research fellows, and clinical and basic science faculty are exploring important questions with research that enhances our excellence in clinical care. We can look forward to another strong year for research at Johns Hopkins All Children’s in 2022.”