Research

With more NIH funding than almost any other medical school-based department of pediatrics in the Northeast, Columbia’s physician-scientists work to address the greatest health threats facing children in the 21st century: obesity, cancer, infectious diseases, diabetes, genetic diseases, heart disease, asthma, and the impacts of prematurity. Our faculty employ cutting-edge research tools such as molecular diagnostics and personalized medicine, and foster wide-ranging and fertile collaborations throughout the university, as well as nationally and internationally. Our goal is to rapidly apply our discoveries to patient care, and to directly benefit the diverse patient community in our region and children around the nation and globe.

Over the past year investigators in the Department of Pediatrics continued our long-standing commitment to innovation through new and ongoing research initiatives. Some 2023 highlights below:

New Center Aims to Lessen the Tragic Consequences of Traumatic Brain Injury

Each year close to half a million children who suffer falls, assaults, motor vehicle or bicycle accidents, or sports injuries are admitted to emergency rooms in the US with a traumatic brain injury (TBI)—and among all types of injuries in young people, TBIs are most likely to cause permanent disability or death. Columbia’s recently appointed chief of pediatric critical care and hospital medicine, Hülya Bayır, MD, has dedicated her career to understanding the brain’s special vulnerability to injury, and what might be done to protect brain tissue in the hours and days after an accident. Through the newly established Columbia Children’s Redox Health Center, she and colleagues are identifying and characterizing a set of key cellular changes that occur following a brain injury, called redox reactions—shorthand for reduction and oxidation. Collaborating with chemists, biophysicists, analytical chemists, and using highly advanced and rapidly evolving tools such as imaging mass spectrometry, Dr. Bayır is detailing the landscape of of redox reactions. This new information will allow her team and others to design novel drug therapies or identify existing, potentially effective medications that may save essential brain cells from destruction. This goal is especially motivating when caring for children who have the misfortune to land in the intensive care unit, she says. “To quote one of my mentors, in pediatrics we treat 20 percent of the population, but they are 100 percent of the future.”

Engaging Community Teens in a Citizen Science Project to Reduce Asthma

As a pediatric pulmonologist, Stephanie Lovinsky-Desir, MD, MS, sees firsthand the association between air pollution exposure in Northern Manhattan and airway inflammation in children living in the community. "Air pollution is a huge factor in child health. In Washington Heights and neighboring communities like the Bronx, where children are exposed to greater concentrations of air pollution, we see higher rates of asthma." Through a research project last summer, Dr. Lovinsky-Desir partnered with Futures Ignite, an organization that advocates for environmental justice, particularly in the Washington Heights community surrounding Columbia University Irving Medical Center. The goal was to help local students measure air quality in their community and use the resulting data to advocate for improvement. "This project is really cool because it's driven by the students," Dr. Lovinsky-Desir says. "We helped them generate data that they can take to city officials and use to advocate to pedestrianize the street adjacent to their school, eliminate street traffic, and beautify the neighborhood," she says. "Working with students who are really excited about being citizen scientists and learning about their environment and their health gives me hope for future generations. These are the people who are going to help to move the needle in terms of health inequity."

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Initiative Brings Experts Together to Diagnose Rare Genetic Disorders

What’s better than a clinical geneticist working to identify the link between a rare mutation and a rare immune disorder? The answer: a group of basic scientists, clinical immunologists, geneticists, rheumatologists, hematologists, and transplanters, all putting their heads together each month to share their expertise, with the same goal. That’s the concept of a new multi-institutional initiative centered at Columbia, NY ROYAL (New York Regional Inborn Errors of Immunity Resource Initiative League). “Physician-scientists caring for patients with rare immune disorders are seeing more and more situations where patients get sequencing done using panels of hundreds and hundreds of genes,” says pediatric immunologist Josh Milner, MD. The results sometimes immediately suggest how the patient should be treated, and may even point to a cure, he says. Together with Dusan Bogunovic, PhD, who will join the Department of Pediatrics this spring, Milner has brought together a group of researchers in the region, each with deep knowledge of one or more specific genes. “No one person can possibly be an expert in 570 different immune problems, so we're crowdsourcing all aspects of this—the genetics, the association between a mutation and a disease, the functional assessment, and the clinical expertise.” The group’s work is now underwritten by an NIH R24 resource grant, which provides funding for the database the group is creating as well as for group members’ time to meet every other week. During the dozen or so NY ROYAL meetings to date, members have assessed two or three cases per session, and hope to increase that to four. “At this pace we could solve a hundred cases a year—that would be pretty exciting,” Dr. Milner says. “What’s become really clear is that we can only do this together.”

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Pathogens’ Metabolic Choices Impact Our Immune Defenses

The immune and metabolic systems are traditionally seen as performing discrete, separate functions in the body’s cells and tissues. Immune system activities protect the body from infection, wounds, and tumors, while metabolic activities produce the energy that fuels cells. Through a growing area of research—immunometabolism—scientists are exploring the crosstalk between the two systems in conditions and diseases ranging from cancer, to autoimmune disorders, to complications of obesity. Sebastian Riquelme, PhD and colleagues have shown that, in some cases of pneumonia, the interplay of immunometabolic activities and antibiotic-resistant “superbugs” Pseudomonas aeruginosa and Staphylococcus aureus in lung tissue can lead to persistent, damaging inflammation, especially in the lungs of people with cystic fibrosis. Complex interactions between the immune and metabolic systems and pathogen itself can cause a “biofilm” to develop in the lungs, a setting in which the bacteria are able to thrive. In most healthy people, by contrast, lung tissue reaches a healthy détente with pathogens, so that the superbugs calm down. Through a growing research portfolio in immunology and immunometabolism, Dr. Riquelme hopes ultimately to understand why some inflammatory reactions to pathogens are benign while others are severe and persistent, and to find ways to tamp down infection severity. He recently received an NIH grant to study how metabolites called nucleotides (best known as the building blocks of DNA and RNA) may modulate inflammation in lung infections. “In this project, we hope to understand how the host’s nucleotide metabolism impacts the persistence of opportunistic superbugs like Pseudomonas and Staphylococcus, which should reveal ways we can prevent or clear these dangerous infections.” Dr. Riquelme says.

Columbia Team Provides Expertise to NIH Pediatric Long COVID Research

Of the more than 700 million people around the world who have had COVID, millions could have long-term, complex health effects. There’s a lot we don’t yet know about long COVID in adults, but it is even less understood in children, including how many have developed the condition and the cause of their symptoms. Part of the National Institutes of Health RECOVER (Researching COVID to Enhance Recovery) Initiative, a nationwide multi-center observational study, is dedicated to studying the condition in children, as well as adults and pregnant people. Through the RECOVER pediatric cohort protocol, children and young adults—both those who have long COVID and others who have not—take part in a number of tests. A team of Columbia researchers was selected by the NIH to help develop and conduct heart and lung testing for some of these children. Aimee Layton, PhD, assistant professor of applied physiologyin pediatrics, is leading the Columbia team charged with interpreting and analyzing results for a specific test called Cardiopulmonary Exercise Testing (CPET), which carefully measures how the lungs, heart, blood vessels, and muscles in children with Long COVID respond to movement. These tests help researchers learn how COVID may or may not affect this group. “Conducting this specialized testing in some children from this study may give us new information that we can link to how they are feeling, which may enable us to better understand what is causing some of their symptoms,” Dr. Layton says. “Many children with long COVID express an inability to run and play with their friends or even take part in gym class. Understanding why this is happening will help us learn how we can help them.”

2023 Innovation Nucleation Fund Awards

The department’s Innovation Nucleation Fund (INF), established in 2020, brings a venture capital approach to great ideas in children’s health research by offering seed funding to innovators with the most promising projects across our mission: advocacy, clinical, education, basic/translational research, and clinical/translational research. Now in its third year, the fund allows our faculty the freedom to pursue innovative and bold ideas that could lead to the next breakthrough for children and their families. Since its inception the department has awarded more than $560,000 through the INF to Columbia physician-scientists researching ways to improve children’s health. In 2023 the following faculty received INF awards:

Clinical/Translational Research Award

• Morgan Finkel, MD, Child and Adolescent Health: Developing optimal approaches to objective infant-caregiver sleep measures in a racial/ethnic minority pediatric patient population

Basic/Translational Research Award

• Sebastian Riquelme Colet, PhD, Infectious Disease: Impact of Ketogenic metabolism on bacterial pneumonia

Clinical Program Award

• Elena Ladas, PhD, RD, Pediatric Hematology, Oncology and Stem Transplantation: A pilot study to establish and evaluate a multidisciplinary clinical research structure for the prevention and management of obesity in children and adolescents with acute lymphoblastic leukemia (ALL) or a brain tumor

Education Program Award

• Dara Steinberg, PhD, Hematology, Oncology and Stem Transplantation: Improving patient educational materials for children with sickle cell disease undergoing curative therapies

Advocacy Program Award

• Emily Mace, PhD, Allergy & Immunology and Rheumatology: Highlighting exceptional researchers in pediatric science

Faculty Award

• Cong Liu, PhD, Department of Biomedical Informatics: Develop and validate a research data warehouse to support observational rare disease study

Faculty Award

• Richard Polin, MD, Neonatology: Ureaplasma spp./mycoplasma hominis and neonatal morbidity/mortality