Innovation and Respect for Every Child and Family
Clinical Care
We're proud to offer exceptional family-centered care, world-class expertise, and the most advanced treatments to infants, children, and adolescents in our community and beyond. We provide the region's greatest depth and breadth of pediatric care through our faculty and collaborators throughout Columbia University Irving Medical Center and NewYork-Presbyterian Morgan Stanley Children's Hospital. Our programs and clinical research are unmatched anywhere in the New York region. This combination is how we've earned the trust of so many families for all of their care needs. Read on to learn more about our notable achievements in 2023.
First FDA-Approved CRISPR Therapy Expands Options for People With Sickle Cell
After an international clinical trial evaluating the use of gene editing to treat patients with sickle cell disease and thalassemia, the FDA has approved the milestone therapy Casgevy (exagamglogene autotemcel) for treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises. “It’s tremendously exciting,” says hematologist-oncologist Monica Bhatia, MD, associate professor of pediatrics at Columbia University Vagelos College of Physicians and Surgeons, who led the Columbia team that treated pediatric sickle cell patients with Casgevy during the multi-center clinical trials. Casgevy is the first therapy approved for use in the United States that employs CRISPR, a gene-editing tool that can make precise changes to a patient’s gene. Dr. Bhatia explains that, “By editing certain cells, we are able to increase the number of oxygen-carrying cells, and decrease pain caused by vaso-occlusive crises and hospitalizations for people with sickle cell disease.” The treatment offers a welcome alternative to bone marrow transplantation to treat the disease. Transplants aren't an option for most patients, and those who do receive a transplant must remain on immunosuppressive drugs to avoid organ rejection. Casgevy is different in that it there is no risk of rejection, because there is no donor. “With gene therapy, you rely on your own cells,” says Dr. Bhatia.
Celebrating Liver Transplantation Milestones
When Chad Glaser donated a portion of his liver to his toddler son Ethan in 2003, he envisioned sharing his many interests with his grown child. The father-son duo celebrated the 20th anniversary of that life-saving procedure with a skiing trip to the Alps this past year. Ethan is just one of the thousands of children whose liver conditions are serious enough that a transplant is the only route to a cure. While Ethan and his family celebrated this meaningful milestone, the NewYork-Presbyterian Center for Liver Disease and Transplantation also celebrated its own: the team performed its 500th pediatric liver transplant in mid-2023. Since its beginning in 1998 the program has become a national leader in the field, and is now among the top five in the country for the number of transplants performed. Great teamwork, innovation, and support made this recent Center milestone possible, noted pediatric hepatologist Dr. Mercedes Martinez, medical director of the Intestinal Transplant Program at the Center for Liver Disease and Abdominal Organ Transplantation at NewYork-Presbyterian. “It is incredibly rewarding to make a life-changing impact and help children lead healthy lives.”
Saving Two Babies Lives With One Pioneering Approach to Heart Transplantation
Mia Skaats and Brooklyn Civil were both born with rare, life-threatening congenital heart defects, and both had spent much of their short lives in the neonatal cardiac intensive care unit (NCICU) at NewYork-Presbyterian Morgan Stanley Children’s Hospital. Both were part of the first-ever domino heart valve transplant in infants. A domino transplant happens when one patient receives an organ transplant and, in turn, donates a healthy organ or healthy parts of an organ to another patient, becoming both a recipient and a live donor. In Mia and Brooklyn’s case, Mia received a full heart transplant, which then made it possible for the healthy valves from her old heart to be placed into Brooklyn’s heart through a partial heart transplant. Making it all happen required the right patients, the right timing, and the tireless teamwork of a multidisciplinary care team that included the transplant service team, child life services, nurses, doctors, the perioperative and surgical teams, and more. “To me, the biggest part of this is that we went from helping one baby to two,” says pediatric cardiologist Dr. Marc Richmond, director of the Program for Pediatric Cardiomyopathy, Heart Failure and Transplantation at NewYork-Presbyterian Morgan Stanley Children’s Hospital, who led the girls’ pre- and postoperative medical care teams. “The number one reason babies die while on a transplant list is because they don’t match with a donor fast enough. So the ability to help more than one child — that’s the holy grail for us.”
New Program Provides Integrated Support for Transplant Recipients
Dr. Paul Sue has a simple analogy for the children he cares for and their families when he talks about the reaction of the immune system to an organ or bone marrow transplant: to the body, a newly transplanted organ looks like a 20-story, neon skyscraper, randomly inserted into a small and tranquil neighborhood. The patient’s natural immune system, acting as the local neighborhood patrol, recognizes it as out of place and a threat to the stable order of things, and fights to eliminate it, leading to rejection. Survival of the organ depends on reducing this vigilant response through drugs that modify the immune system’s natural reaction, allowing the organ to remain. The costs of this lowered vigilance, however, are previously suppressed infections that begin to slip through and can threaten the entire neighborhood. Over time, as the body becomes more accepting of the organ, less rejection medication is needed and these risks of infection begin to decline. But they always persist in some form, and patients typically require transplant medications for the rest of their lives. Dr. Sue recently joined Columbia as medical director of the newly established PITCH (Pediatric Transplant & Immune Compromised Host) program at CUIMC/NYP, one of the largest transplant centers in the country. “PITCH ID allows us to integrate and work hand in hand with our surgical, medical, and transplant teams to better focus on the unique infectious disease risks of the transplanted and immunocompromised child,” Dr. Sue says. “Our goal is to recognize and treat these opportunistic infections in a well-coordinated and timely fashion, so that viruses, fungi, and bacteria don't get in the way of the second chance our kids have to live out their fullest potential.”
Notes From the Transplantation Frontlines
Primary immune deficiencies (PID) and primary immune regulation disorders (PIRD) are rare genetic diseases that disable the body’s immune system. Because the immune system has trouble functioning or regulating itself, children with these conditions tend to develop problems such as inflammation, autoimmune conditions, and allergic conditions, and have difficulty fighting off infections. At the same time that genetic testing is revealing the underlying genetic causes of many of these diseases, treatment options are expanding with new targeted therapies and stem cell transplantation approaches. Bone marrow and stem cell transplant specialist for Olatundun Williams, MD is an innovator in transplant approaches for PIDs and PIRDs in children. “Over the past decade or so, we have witnessed the development of targeted therapies for some of these disorders,” she says, “but transplantation is something that we can offer these patients beyond the limited arsenal of medicines, and, in some cases, has the potential to be a cure.”