Department of Pediatrics Annual Report – 2020

Research

The top department of pediatrics in the NY region in NIH research funding

The Department of Pediatrics has a long-standing commitment to innovation. Columbia researchers have been working to improve the health of children since the medical college’s founding as part of King’s College in 1770. At that time university physicians studied deadly conditions like diphtheria and “blue baby” syndrome. Since then, generations of Columbia pediatricians have made seminal contributions to the most prevalent and devastating diseases that impact children.

With more NIH funding than any other department of pediatrics in the Northeast, our physician-scientists are addressing the greatest threats facing children in the 21st century: obesity, cancer, infectious diseases, diabetes, genetic diseases, heart disease, asthma, and the impacts of prematurity. Our faculty emphasize contemporary research themes, including molecular diagnostics and personalized medicine, and foster wide-ranging and fertile collaborations throughout the university, nationally, and internationally. We strive to rapidly move our discoveries to patient care to directly benefit our diverse patient community in the New York region and children around the nation and globe. Discovery and innovation reside in the DNA of our department.


A human egg being injected with CRISPR/Cas9
A human egg being injected with CRISPR/Cas9 - Egli Lab

Major Areas of Research and Specialized Programs

  • Genetics and genomic medicine
  • Nutrition, metabolism, and gastrointestinal disorders
  • Cardiovascular and circulation
  • Lung health and breathing
  • Regenerative medicine, injury, and repair
  • Behavior, development, and mental health
  • Pharmacology and drug development
  • Infection and immunity
  • Cancer and blood disorders
  • Health services, prevention, and environment

Below are just a few highlights of the extraordinary investigative work in the department. The overview of our clinical research and translational and laboratory-based research projects provide a snapshot of our overall research enterprise.


Clinical Research

Clinical observations and trials, designed to answer a specific question about the safety and efficacy of a new drug, device, or procedural intervention, remain the hallmark of patient-directed research. Our department’s clinical research is multifaceted, all encompassing, patient-centric, and conducted by every learner and faculty member.

Heart Disease

Clinical research in pediatric cardiology and cardiac surgery investigate pharmaceuticals and devices across the spectrum of heart illnesses and encompass two overarching themes:

  • Congenital heart disease affecting fetuses, children, and adults
  • Non-inborn, organic cardiac diseases of childhood and adolescence

Newborn Care

The overarching focus of neonatal investigators is the clinical physiology of the cardio-respiratory system in premature infants. Ongoing investigations include:

  • Respiratory and hemodynamic effects of continuous positive airway pressure
  • Non-invasive nitric oxide to treat hypoxemic respiratory failure
  • Pre- and postoperative care of neonates with congenital heart diseases

Cancer and Blood Disorders

Our investigators maintain a large number of trials through national and international children’s oncology consortia:

  • Children’s Oncology Group
  • Dana Farber Cancer Institute
  • Leukemia Consortium for Therapeutic Advances in Childhood Leukemia & Lymphoma
  • National Marrow Donor Program
  • Pediatric Cancer Foundation Developmental Therapeutics Phase 1 Program. Through this program we:
    • Are one of only 20 centers in the country
    • Are the only site in the New York-New Jersey-Connecticut area
    • Are supported by the National Cancer Institute-sponsored Children’s Oncology Group Consortium
    • Provide experimental cytotoxic drugs, biologics, and immunotherapies

Our Precision in Pediatric Cancer through Sequencing (PIPseq) Initiative connects patients with innovative therapies based on their precise molecular-genetic results.

We are among the first centers in the nation to provide gene therapy autologous transplantation for patients with sickle cell anemia and thalassemia.

Lungs and Breathing

Physician-investigators in the Division of Pulmonology participate in the Cystic Fibrosis Foundation’s Therapeutics Development Network. Additional clinical studies include:

  • Controlling asthma among inner-city minority children
  • Evaluating risk factors for sleep problems and methods to improve sleep in children

Basic and Translational Research

Primary Atopic Disorders

“Although allergic disorders are extremely common in children, their immunologic and genetic complexity has only recently been appreciated,” notes Joshua Milner, MD, whose laboratory probes the basic immunological mechanisms that drive allergic diseases and immunological dysfunction. His research led to the discovery of primary atopic disorders, a group of genetic conditions that predispose individuals to develop allergic conditions, ranging from severe hives to eczema to food allergies.

Hormonal Factors and Newborn Neurodevelopment

Anna Penn MD, PhD, and colleagues use genetic models to understand the hormonal factors that contribute to normal neurodevelopment and the impact of their absence following premature birth or placental compromise. “We have learned that disruption of placental function, secondary to a multitude of events—infection, malnutrition, genetic abnormalities, and preterm birth—alter the hormonal environment of the newborn brain,” Dr. Penn says. Such alterations increase the developing brain’s susceptibility to damage that leads to cerebral palsy, developmental delay, and other lifelong problems.

human natural killer cell migrating on fibronectin
Human natural killer cell migrating on fibronectin - Mace Lab

Natural Killer Cells and Novel Immune Diseases

International leaders in pediatric primary immunodeficiency and the immunobiology of human natural killer cells, Jordan Orange, MD, PhD, and Emily Mace, PhD, collaborate to translate underlying biological mechanisms of immunodeficiency disorders into clinical application. “Natural killer cells are there—always ready—to protect us against danger,” Dr. Orange says. “When they malfunction, patients typically develop severe disease and suffer from profound and often lethal susceptibility to viral infection and cancer.”

The Fight Against Viral Diseases

Lower respiratory tract infections cause more illness and death in children worldwide than any other factor. The laboratories of Anne Moscona, MD and Matteo Porotto, PhD are known for discovering the biological mechanism for the first step of respiratory viral infection—fusion of virus into lung cells—and using that science to design antivirals that prevent illness. Drs. Moscona and Porotto note that, "our response to SARS-CoV-2 was to use our knowledge to develop a nasal-spray antiviral to prevent transmission of SARS-CoV-2 and future strains. Our goal is to apply our research to protect children from lung viruses that affect children everywhere."

Developmental Origins of Resilience

The Developmental Origins of Resilience (DOOR) laboratory, led by Dani Dumitriu, MD, PhD, is dedicated to identifying mechanisms for individual variability in response to stress, depression, and anxiety. Dr. Dumitriu says, “Using animal models and applying state-of-the-art whole-brain imaging tools and super-resolution imaging of dendritic cells we have discovered preexisting differences in the neuro-circuit structure-function between animals who are susceptible to stress and those who are resilient.”

Digital Health Interventions

Melissa Stockwell, MD, MPH, and her team in Columbia’s Center for Children’s Digital Health Research program focus on interventions to promote vaccination among underserved children and adolescents. Their research tools include translational health IT interventions, and patient-centered communication technologies like text messaging. “We are designing interventions to overcome personal, provider, and community barriers to vaccination,” Dr. Stockwell says. “We’re also working to demonstrate how integrated electronic vaccination data sources can improve patient care and population health.”

DISCOVER: Genetic Basis of Human Disease

Wendy Chung, MD, PhD, and her research team investigate the genetic basis of a variety of diseases, including obesity, type 2 diabetes, heart disease, endocrinopathies, congenital diaphragmatic hernias, cleft lip/cleft palate, neurological and neuromuscular disorders, inherited metabolic conditions, and breast and pancreatic cancer, and a new paradigm for early diagnosis and intervention for spinal muscular atrophy. Through the DISCOVER Program, she uses cutting-edge research to diagnose rare diseases and develop innovative and novel individualized treatments for patients.

Brain Tumors: Novel Drug Delivery

Stergios Zacharoulis, MD, leads the newly created Brain Tumor Technology Research program, an initiative created to improve clinical outcomes in pediatric brain tumors, the leading cause of death for children with cancer. “My colleagues and I use a novel drug-delivery method that includes a complex combination of biomedical engineering, bioinformatics oncology, and neurosurgery to potentially deliver drugs to brain tumors more efficiently,” Dr. Zacharoulis says. His team has developed a unique convection-enhanced device that delivers intratumoral infusions of medicine via a wireless subcutaneous pump connected to a catheter implanted within the brain tumor.

Pulmonary edema in mouse lung
Pulmonary edema in mouse lung - Rebecca Hough, MD, PhD

Lung Injury and Repair

In their studies of lung injury and repair mechanisms, Danielle Ahn, MD, and Rebecca Hough, MD, PhD, are investigating, respectively, host-adapted mechanisms of impaired bacterial clearance in the lungs and how acute lung injury signals are communicated from alveoli to lung capillaries. Using a murine model of pneumonia and bacterial mutagenesis, Dr. Ahn says, “My goal is to identify novel treatments to modify and effectively resolve an infection before it develops into a life-threatening illness.” Dr. Hough focuses on the early events of lung injury that cause acute respiratory distress syndrome (ARDS). Dr. Hough says, “ARDS can lead to long-term lung damage or death, and I hope this work opens the door to new molecular targets for therapy.”


INF Awardees 2020
Recipients of INF awards, from left, are Sumeet Banker, Jennifer Woo-Baidal, Andrew Geneslaw, Julia Wynn, and Rebecca Hough. Not pictured: Michael DiLorenzo. (Photo: Charles Manley)

Children’s Health Innovation Nucleation Fund

Established with contributions to the Department of Pediatrics from private donors, the Innovation Nucleation Fund (INF) brings a venture capital approach to supporting new research in children’s health. The INF awards of $10,000-$50,000 provide talented faculty the freedom to pursue innovative, bold ideas in research that could lead to the next major breakthrough in pediatrics. In the program’s inaugural year, the department awarded six grants to the following young investigators:

  • Andrew Geneslaw, MD, Division of Critical Care and Hospital Medicine, Faculty INF Award for “The Incidence of Childhood Cognitive and Neurodevelopmental Dysfunction After Respiratory Failure.”
  • Sumeet Banker, MD, MPH, Division of Child and Adolescent Health, Advocacy Award for “Improving Comprehension and Experience of Limited English Proficient Population on Family Centered Rounds.”
  • Julia Wynn MS, CGC, Division of Molecular Genetics; Co-I: Priyanka Ahimaz, MS, CGC; Ilana Chilton, MS, CGC; Emily Griffin, MS, CGC; Rebecca Hernan, MS, CGC, Education Award for “Developing an Interactive, Adaptable Video Educational Tool for Genomic Research.”
  • Rebecca Hough, MD, PhD, Division of Critical Care and Hospital Medicine, Basic/Translational Research Award for “The Hippo Pathway in Mechanical Stretch-Induced Acute Lung Injury.”
  • Michael DiLorenzo, MD, MSCE, Division of Pediatric Cardiology, Clinical Research Award for “Lymphatic Imaging and Intervention Program at Columbia University Irving Medical Center/Morgan Stanley Children’s Hospital.”
  • Jennifer Woo-Baidal, MD, MPH, Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Co-I: Dodi Meyer, MD, Clinical-Translational Award for “Food FARMacia: Evaluating an Innovative Approach to Reduce Food Insecurity and Prevent Childhood Obesity in the First 5 Years of Life.”

The department has received applications for the INF’s second year of awards, which will be announced in early 2021.


COVID-19 Research

Physician-scientists in the Department of Pediatrics are turning their expertise to the myriad questions raised by SARS-COV-2 and its impact on infants, children, and adolescents. Researchers in almost every division in our department are collaborating with experts throughout Columbia’s departments and schools, and at centers around the country and world.

Learn more about our department’s COVID-19 research

Noteworthy is the groundbreaking, critical research, funded in small part by the department, of Drs. Moscona and Porotto who have developed lipid-peptide molecules designed to prevent COVID-19. These antivirals can be administered via nasal drops, are nontoxic, and have good half-life in the lungs. Drs. Moscona and Porotto plan to conduct a trial using nasal drops or spray to prevent infection of health care workers.