Children’s National Research Institute Academic Annual report 2019-2020
From the Chief Research Officer
150 Years Stronger Through Discovery and Care
This year, we celebrate a unique birthday in the life of Children’s National: 150 years of providing pediatric care, research and community commitment to the children of Washington, D.C., and the nation.
As one of the nation’s oldest children’s hospitals, our history is distinguished by our steadfast dedication to keeping the needs of children and their families at the heart of all we do and to make our children healthy, stronger and more resilient.
This birthday year, however, is now a somber occasion, given the current state of the world and the devastating impacts of the pandemic of COVID-19. However, it is the strong history of groundbreaking research here at Children’s National coupled with the world-renowned expertise of our faculty and staff that give me hope for better days on the horizon.
Our inaugural research program in 1947 began with a budget of less than $10,000 for the study of polio — a pressing health problem for Washington’s children at the time and a pandemic that many of us remember from our own childhoods. Today, our research portfolio has grown to more than $75 million, and our 314 research faculty and their staff are dedicated to finding answers to many of the health challenges in childhood. Obviously, the most pressing health challenge as we write is that of COVID-19 and the virus SARS-CoV-2. In less than 6 months’ time, the research team at Children’s National has taken their expertise and focus in this new direction to enhance global understanding of how this previously unknown threat impacts people of all backgrounds and ages. It is very possible that our talented and energetic group can contribute vital information, or perhaps even help find a breakthrough treatment, that will be the difference between life and death for hundreds of thousands of people.
We are poised to do this work. In the past few years, major breakthroughs have been occurring within our walls, including:
- Understanding the molecular and genetic basis for rare hereditary diseases — such as urea cycle disorders, which can be deadly to children in their first week of life — and then finding better ways to identify those issues in utero and be ready to protect a newborn from their damaging effects as soon as possible after birth.
- Illuminating how the brain’s white matter plays a crucial role in growth and development and examining mechanisms that might trigger regrowth and repair after oxygen deprivation leads to white matter damage.
- Developing novel cell-based immunotherapy techniques that lead disease-fighting T cells from donors to take on and fight relapsing cancers and devastating viral infections.
- Validating models of care that help children and their families find better ways to manage chronic conditions such as asthma by co-locating teaching and medical care in a single location, such as the emergency department, which can reduce return visits for urgent treatment down the road.
- Leading the development of child-sized surgical devices and other pediatric-focused innovations, including minimally invasive pacemakers, cellphone-based software to objectively measure pain and three-dimensional models of critical congenital heart disease to permit simulation of planned surgeries.
Those research successes and innovations have not happened in one laboratory or through one clinical research study. Instead, it’s the unique ability at Children’s National for physicians and scientists to collaborate, often joining forces across institutions and governmental agencies to plan and carry out studies.
Making sure these activities occur in a collaborative manner is key to accelerating the timeline for getting new medical advances to the children who need them, both in our region and — truly — around the world.
This year, 2020, also marks the start of a new physical chapter for our research and clinical care activities working together: the opening of Phase 1 of the Children’s National Research & Innovation Campus. This first-of-its-kind endeavor is an undertaking that will grow into a true ecosystem to nurture pediatric innovation from discovery in the laboratory through commercialization of products — products inspired by the needs of children and tailored to their bodies. We believe that this ecosystem is the structure that will drive the next generation of research and clinical care and keep our institutions at the forefront of research into devastating diseases both well-known and new, like COVID-19.
It seems unbelievable that what started as a 12-bed, volunteer-led orphan hospital founded just after the Civil War could grow into the global leader of advanced pediatric health care that we have become. Our position today as the number six children’s hospital in the country, home to the nation’s number one neonatal intensive care unit (NICU), and one of the top-funded pediatric research institutions in terms of National Institutes of Health support, however, has only been possible thanks to our institution’s dedication to providing exemplary care for children. At the same time, we have maintained a focus on conducting exemplary pediatric research and providing world-class training and education in pediatric medicine.
We are proud of the synergy between research, education, innovation and clinical care at Children’s National. Many of our clinical leaders also have strong scientific backgrounds and are actively engaged in funded research. This fact also makes Children’s National one of the best places in the country to train as a physician, nurse or other health care professional. Not many places in the United States can offer an opportunity for an aspiring pediatric researcher or clinician to learn in this interprofessional environment; we think that this is the model for the future.
One example of this model is our Children’s National Research & Innovation Campus It will house our Genomics and Precision Medicine initiative as well as the clinical and research activities within our Rare Disease Institute, the Clinical Molecular Diagnostic Laboratory and a new outpatient care center for the families of Ward 4. We’ve also secured some exciting new partners to live and work with us on the campus — Johnson & Johnson Innovation’s JLABS @DC (an accelerator-incubator for start-up companies focusing on pediatric care) and Virginia Tech, which will bring a biomedical focus to pediatric brain tumor research to the site.
While research and innovation hubs are finding success across the country, ours has one critical distinction: it focuses on diseases and conditions affecting the health of children. This distinction is enhanced by the close marriage of research and clinical care working side by side.
Our work with our academic partner, the George Washington University, also has continued to thrive, and we are excited about what the future holds for our ongoing research partnerships and connections across that prestigious institution, including our joint Clinical and Translational Science Institute (CTSI-CN), Center for AIDS Research (CFAR), the District of Columbia Intellectual and Developmental Disabilities Research Center (DC-IDDRC) and cancer program as well as academic relationships with the School of Medicine and Health Sciences, the School of Public Health and many others.
This year’s report spotlights some of our recent work across the organization and around the world which was well underway and continues despite the global spread of COVID-19, including:
- The latest developments in the growth of the Children’s National Research & Innovation Campus. Research that brings diverse reference genomics to bear on our understanding of disease and how it affects the people of Central Africa and their descendants.
- Explorations of philanthropic, family and community engagement in the research and clinical hunt for better treatments and support of patients with polycystic kidney disease (PKD).
- The organization’s creation of networks, collaborations, partnerships and consortia with like-minded clinicians, scientists, governmental agencies — especially a flagship partnership between the Clinical and Translational Science Institute at Children’s National and the NIAID — as well as communities locally, nationally and around the world to drive treatment breakthroughs for children no matter where they live.
- Efforts in clinical and research neurosciences to educate, train, support and mentor the best pediatric clinicians, scientists and clinician-scientists possible — from every background and from around world.
- Leading cellular immunotherapy clinical trials that are having lifesaving results for children with relapsing refractory solid tumors.
- The National Capital Consortium for Pediatric Device Innovation and its efforts to further encourage the development of devices and treatments that take into account the specific needs of children and are designed for a still-growing body.
- Continuing to build our reputation as a premier location for medical education by bringing the key tenets of our world-class pediatric residency program to every aspect of medical education across the organization.
We hope you enjoy reading these highlights; they are only the tip of the iceberg for the broad range and depth of research and innovation work going on within the Children’s National Research Institute. You can read more, and sign up for e-newsletters on specific pediatric topics, at Innovation District.
As we celebrate our sesquicentennial of providing the best care available for children, we eagerly look forward to what the next decades hold in our quest to make sure that all children grow up stronger, even in the face of great medical and scientific challenges that lie ahead—both known and unknown.
Vittorio Gallo, Ph.D.
Chief Research Officer, Children’s National Hospital
Scientific Director, Children’s National Research Institute
Wolf-Pack Chair in Neuroscience
Alpha Omega Alpha Medical Honor Society
Associate Dean for Child Health Research
George Washington University School of Medicine and Health Sciences