From rare diseases to long COVID: A decade of discovery
Since 2017, the collaboration has helped Children’s National investigators launch clinical studies, enroll more than 2,300 participants, establish a biorepository with nearly 67,000 samples and contribute to dozens of scientific publications.
For nearly a decade, researchers at Children’s National and the National Institute of Allergy and Infectious Diseases (NIAID) have advanced discoveries in immunology, infectious diseases, allergy and neuro-immune disorders while expanding research opportunities for children and families.
Since 2017, the collaboration has helped Children’s National investigators launch clinical studies, enroll more than 2,300 participants, establish a biorepository with nearly 67,000 samples and contribute to dozens of scientific publications.
“Transformative discoveries rarely happen in isolation,” says Catherine Bollard, MBChB, MD, senior vice president and chief research officer at Children’s National. “This collaboration has allowed our investigators to ask bigger questions, study rare and complex pediatric diseases at scale and create opportunities for children and families to participate in research that is shaping the future of pediatric medicine. The impact extends well beyond individual studies. We are building the scientific foundation for the next generation of treatments and cures.”
Dive deeper
A major focus has been primary immunodeficiency disorders, rare conditions that affect the body’s ability to fight infection. Children’s National researchers have identified previously unknown inborn errors of immunity, including POLD1 deficiency and FOXI3 deficiency, while improving diagnosis and supporting evaluation of therapies and vaccines in vulnerable children.
The collaboration has also strengthened leadership in pediatric infectious disease research at Children’s National. The Pediatric COVID Outcomes Study (PECOS) enrolled more than 1,000 children and adolescents to study the long-term effects of COVID-19. Researchers completed nearly 4,000 longitudinal visits including cardiac, pulmonary, radiographic, psychological and developmental evaluations, and a biorepository of specimens, creating one of the most comprehensive pediatric datasets on SARS-CoV-2 infection.
Investigators found no evidence that COVID-19 negatively affects heart function in children and adolescents, even among those reporting fatigue, chest pain or palpitations. The study also characterized persistent symptoms after infection and their trajectory across different age groups, adding to understanding of pediatric long COVID and its impact on quality of life.
Researchers have also expanded work on pediatric Lyme disease, including recovery after treatment for all stages of Lyme and lingering symptoms. Early findings suggest most children recover fully, while some may experience more prolonged symptoms and benefit from additional monitoring and support.
Advancing research
In asthma research, investigators are studying immune mechanisms that contribute to persistent disease. Recent findings suggest children with persistent asthma have lower levels of regulatory B cells than healthy children, while patients receiving biologic therapies show levels more similar to healthy controls. Lower regulatory B cells contribute to increased allergic inflammation and likely worsening asthma. Researchers are also examining pediatric neuroimmune disorders through the Natural History Study of Pediatric-Onset Neuro-Immune Conditions with Discovery of Dysregulated Molecular And Genetic Pathways (NI-MAP), which seeks to elucidate the pathogenesis of patients with both known and undifferentiated neuroinflammatory disease, including their clinical, neurologic, immunologic, genetic and metabolic/endocrinologic characteristics.
Training the next generation
The partnership has also created training opportunities for physician-scientists, with dozens of fellows completing specialized experiences in clinical care, research and mentorship. More than 70% of graduates have gone on to careers in academic medicine, government or regulatory agencies.
What comes next
The collaboration has strengthened large-scale pediatric research through streamlined regulatory pathways, electronic consent tools, shared workflows and governance structures.
“What excites me most is how this work continues to grow,” Dr. Bollard says. “The infrastructure, expertise and collaborative culture we’ve built are helping Children’s National tackle some of the most challenging questions in child health today while training the physician-scientists who will lead the field tomorrow.”
Together, these discoveries are improving care today while laying the groundwork for tomorrow’s breakthroughs.
