Spotlight on Innovation

Columbia has long been a leader for innovation and transformative care, thanks in no small part to our culture of collaboration and teamwork. These select programs highlight cutting-edge research and clinical care that leverage the expertise and experience from across the Department of Pediatrics, NewYork-Presbyterian Morgan Stanley Children's Hospital, and Columbia University Irving Medical Center (CUIMC).

Cardio-Oncology Program

Long-term survival has increased in children with cancer, but at the same time cardiovascular issues have become more prominent among survivors. These include cardiac dysfunction, hypertension, atherosclerosis, pericarditis, and significant cardiopulmonary deconditioning, all potentially resulting from chemotherapy or radiation therapy, or in some cases from the primary disease itself. At our center, the divisions of Cardiology and of Hematology, Oncology, and Stem Cell Transplant are working closely to develop a pediatric cardio-oncology program that will better serve children and young adults with hematologic or oncologic conditions as well as those who require stem cell transplantation.

The first of its kind in the New York area, the program will help identify cancer survivors at risk for cardiovascular complications and will provide management with state-of-the-art heart failure/cardiomyopathy services, cardiac imaging, cardiopulmonary exercise testing and rehabilitation, electrophysiologic testing and intervention, and other catheterization-based minimally invasive procedures. Through this collaboration of dedicated specialists, the program aims to improve care for these vulnerable patients and to establish the division as an international leader in the evolving field of pediatric cardio-oncology.

Center for Host-Pathogen Interaction

The Center for Host-Pathogen Interaction (CHPI) is a collaboration between the laboratories of Anne Moscona, MD, and Matteo Porotto, PhD. Drs. Moscona and Porotto together conduct basic research on paramyxoviruses, RNA viruses transmitted by airborne droplets, which are responsible for acute respiratory illnesses prevalent in childhood. Their investigations draw from strategies and methods of molecular biology, cell biology, biophysics, immunology, computational biology, structural biology, and virology and use animal models of human disease to answer questions about viral infection.

The Moscona-Porotto team has discovered the critical roles of the viral receptor binding protein in activating the viral fusion process during infection. The recognized mechanism of fusion activation has led to promising targets for interfering with the viral entry process, which are being evaluated in animal models of disease. These antiviral approaches have provided valuable tools for furthering the understanding of basic mechanisms of infection. The team's infection prevention findings may apply to measles, as well as parainfluenza viruses, influenza viruses, and other emerging lethal viruses such as the Ebola and SARS-CoV-2.

Learn More About the Center for Host-Pathogen Interaction (CHPI)

Childhood Obesity Program

The Columbia Pediatric Obesity Initiative, led by Jennifer Woo Baidal, MD, John Rausch, MD, and Elena Ladas, PhD, aims to address pediatric obesity throughout the life course by providing multidisciplinary care, research, education, and advocacy, with a focus on diversity, equity, and inclusion. The initiative includes experts in pediatric gastroenterology, endocrinology, nutrition, mental health, surgery, and other specialties. Program members provide comprehensive care to patients and also perform research to uncover the genetics and underpinnings of overweightness. Projects include:

• Building Optimal Lifestyles Daily (BOLD), an intensive clinical program aligned with evidence-based interventions using a hybrid model with both in-person and virtual video components 
• The Child Research in Obesity Prevention (CROP) lab, which uses clinical, public health, and patient-oriented data to develop and test interventions aimed at prevention of racial, ethnic, and socioeconomic health disparities in obesity and related complications
• The Genetics of Severe Early Childhood Obesity (GECO) study, a clinical and genomic investigation designed to investigate monogenic causes of severe early childhood obesity

Columbia Center for Children's Digital Health Research

The Columbia Center for Children’s Digital Health Research (CDHR) maximizes digital communication and emerging technologies to improve children’s health here in New York, across the country, and around the world by fostering the design, operationalization, implementation, and evaluation of digital health research that has a direct impact on child and population health. Led by Melissa Stockwell, MD, MPH, CDHR harnesses the potential of well-established, widely used technologies such as text messaging and artificial intelligence (AI) to design, implement, and evaluate digital health research. CDHR areas of engagement include mHealth, eHealth, telemedicine, AI, electronic health records, clinical decision support, personal health records, and social media.

To support CDHR’s mission, the center provides the following key services: 

• Consultation and collaboration on new research
• Guidance for evaluation and implementation of digital health tools
• Hands-on training for researchers

Learn More About the Center for Children’s Digital Health Research

Congenital Diaphragmatic Hernia Program

Congenital diaphragmatic hernia (CDH) is a rare congenital condition in which the diaphragm does not form properly, allowing gastrointestinal organs to move up into the chest, preventing normal function of the lungs and often the heart. CDH may be diagnosed prenatally by an ultrasound and other tests at Columbia’s Center for Prenatal Pediatrics, a multidisciplinary center renowned for expertise in  high-risk births and complicated newborn conditions. Babies in need of heart-lung life support prior to surgery benefit from our CDH-specific extracorporeal membrane oxygenation (ECMO) protocol. After hernia closure, children with CDH require long-term monitoring.

Columbia’s CDH Program brings together pediatric experts in gastroenterology, surgery, neonatology, cardiology with special expertise in pulmonary hypertension, lung dysfunction, nutrition, feeding problems, and developmental, neurological, orthopedic, and psychological issues.

Learn More About the Congenital Diaphragmatic Hernia Program

GUARDIAN Initiative

Newborn screening (NBS), the screening of all newborns for select conditions shortly after birth, detects medically actionable conditions in presymptomatic newborns, thereby reducing morbidity and mortality. NBS by genetic sequencing (GS) is intended to expand present NBS methods to provide screening for more conditions, enabling early treatment and intervention and improving outcomes for more children. In particular, this technology has the capacity to screen for susceptibility to neurodevelopmental conditions that are not detectable by current NBS platforms. Members of the Division of Clinical Genetics have launched the GUARDIAN study, a program to perform newborn genetic sequencing on dried blood spots already collected as part of the New York State newborn screening mandate. The GUARDIAN initiative is a pilot study of expanded GS NBS across New York City hospitals for 200 conditions not included in the current standard NBS panel of approximately 50 conditions.

Inflammatory Bowel Disease Program

The Inflammatory Bowel Disease (IBD) Program offers patients with early onset and complex IBD outstanding diagnostic, medical, nutritional, and surgical management. Neal LeLeiko, MD, PhD, medical director of the program, collaborates with Bo Shen, MD, a leading adult IBD interventionalist, and pediatric gastroenterolgist Joseph Picoraro, MD, to offer patients advanced diagnostic and therapeutic techniques that few other institutions can provide.

Members of the program work closely with pediatric and colorectal surgeons and with immunologists Josh Milner, MD, and Ramsay Fuleihan, MD, who provide unparalleled expertise in therapeutic immunology. In addition, patients have access to Columbia experts in celiac disease and eosinophilic gastrointestinal disease, including eosinophilic esophagitis, and in the liver complications of IBD, such as primary sclerosing cholangitis.

A highlight of the program is a systems-based, quality improvement initiative that promotes patient directed infusion-based therapies including rapid infusion, home infusion, and COVID-safe practices. IBD team members participate in and lead research network and multicenter clinical trials with other pediatric IBD experts from across the country.

Learn More About the Inflammatory Bowel Disease Program

Liver and Multiorgan Transplant Center

Founded in 1998, the Liver and Multiorgan Transplant Center at Columbia Children’s Health is one of the first and largest programs in the country. The team performs more transplants than most other hospitals in the country and offers innovative treatments such as a fully laparoscopic hepatectomy. The medical team, led by Mercedes Martinez, MD, and Steve Lobritto, MD, is the top-ranked pediatric transplant center in New York State by volume since 2008 and the fourth ranked liver transplant program in the U.S. 

Embedded within the Liver and Multiorgan Transplant Center are a number of key clinical enterprises:

• Living Donor Program: We have pioneered and perfected the use of this innovative procedure for both children and adults, helping to increase survival rates and reduce the wait for transplants. 
• Pediatric Liver Program: Our team, which includes three board-certified pediatric gastrointestinal transplant specialists, provides nonsurgical care in addition to surgery and transplantation.
• Intestinal and Multiorgan Transplant Program: Our doctors have contributed significant advances to procedures in which surgeons transplant several organs at once, giving patients with serious intestinal malformations or infections hope for a healthy future.
• Comprehensive Nonalcoholic Fatty Liver Disease (NAFLD) Program: Our nationally recognized program uses a variety of tools, including genetic testing, imaging, experimental therapeutics, and behavior modification, for long-term care of patients with NAFLD and nonalcoholic steatoheptitis (NASH).
• Columbia’s Transplant Clinical Research Core: This multidisciplinary intellectual focus for all transplant researchers at CUIMC brings basic science, translational science, clinical research, and multidisciplinary clinical departments together to seamlessly translate advances in the lab into clinical care.

Learn More About the Liver and Multiorgan Transplant Center

Lymphatic Imaging and Intervention Program

The Lymphatic Imaging and Intervention Program is a new initiative geared to patients with congenital heart disease (CHD), particularly those who have undergone single ventricle palliation. Disordered lymphatic circulation may contribute to other serious complications such as chylous effusions, plastic bronchitis, and protein-losing enteropathy (PLE), which can be extremely difficult to treat and result in significant morbidity, frequent and prolonged hospitalizations, and increased mortality risk.

Recent innovations in MRI-based lymphatic imaging and percutaneous lymphatic embolization techniques have demonstrated impressive early success in managing these conditions and represent a “new frontier” in caring for patients with single ventricle heart disease. Co-directed by Michael DiLorenzo, MD, and Matthew Crystal, MD, from pediatric cardiology and interventional radiologist Sheryl Tulin-Silver, MD.

The Columbia team at NewYork-Presbyterian Morgan Stanley Children’s Hospital is the only group in the New York metropolitan region with the resources and expertise to develop a multidisciplinary imaging and intervention program benefitting CHD patients as well as those with non-cardiac lymphatic abnormalities.

National Organization for Rare Disorders Center of Excellence

The National Organization for Rare Disorders (NORD) is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

In 2021 NORD selected CUIMC with NewYork-Presbyterian Hospital as a Center of Excellence, joining a national network of 31 centers dedicated to improving treatment and care for people with rare diseases. Wendy Chung, MD, PhD, physician-scientist, clinical geneticist, and principal director of the center says, “This is an important time in medicine, when we have the ability to identify more patients with rare diseases, identify what causes the disease, and use precision medicine to find ways to treat them.”

Columbia investigators will be able to share the collective power of our multidisciplinary clinical expertise, translational research infrastructure, and genetic testing/counseling to help more patients living with rare diseases, including spinal muscular atrophy, mitochondrial disorders, rare cancer syndromes, immunological disorders, and cardiac disease.

Read More About Columbia and NORD

Sickle Cell Anemia Gene Therapy Program

In sickle cell anemia (SCA), an abnormal beta-globin (HbB) gene triggers the production of rigid, misshaped red blood cells with decreased capacity to carry oxygen to the tissues. Complications of SCA range from chronic pain to organ failure and sudden death. In the mid-1980s investigators reported using allogeneic stem cell transplantation (SCT) to replace the HbS with normal hemoglobin production in patients with severe SCA. Today, physician-scientists at Columbia Children’s Health, led by Monica Bhatia, MD, have taken SCT to the next level: using gene therapy strategies to definitively “cure” the disease.

Dr. Bhatia and her team are participating in a national collaborative study of gene therapy for SCA, CLIMB: SCD-121, and CLIMB: THAL-111 for patients with severe b-Thalassemia. In both studies, stem cells are collected from the patient and shipped to a central CRISPR-Cas9 gene editing facility. The patient receives myeloablative conditioning followed by infusion with the genetically edited material (CTX001). They are then monitored for engraftment, hemoglobin production, hemolysis, HbF and F-cell expression, and signs and symptoms of disease. Dr. Bhatia says, “We are optimistic that we have uncovered a potential cure for sickle cell anemia and b-Thalassemia, two genetic diseases that previously had significant morbidity and mortality.”

Read More About the CLIMB Clinical Trial