NEC is the most common and lethal gastrointestinal disease in preterm infants. It is caused by bacteria invading the small intestine, causing infection and inflammation that ultimately destroys the wall of the bowel. Once the disease takes hold, surgery to remove the dead tissue is the only treatment option available. This leaves the infant with insufficient intestine to absorb nutrients for growth, known as short bowel syndrome. Because its origins are still unknown, no preventative strategy has meaningfully reduced the rate of NEC.
Phil Tarr, MD, the Melvin E. Carnahan Professor in Pediatrics and pediatric gastroenterologist, possesses a passion for finding a cure for NEC. With funding from the CDI, Dr. Tarr teamed up with equally passionate Children’s neonatologist Barbara Warner, MD, to collect fecal samples from premature babies. Their goal was to identify and quantify the differences between the microbial communities of infants who develop the illness and those who do not.
After hearing Dr. Tarr speak at a conference, Brigida Rusconi, PhD, knew she had found a new home to pursue her research interests. She applied for and received a fellowship to come to St. Louis and join Dr. Tarr’s lab. Now, with CDI funding of her own, she will employ the bank of fecal samples to look for markers that could be used to alert clinicians that an infant is at risk of developing NEC. She is most interested in describing the role a certain type of lipid plays in that development.
Like Dr. Rusconi, Kristin Seiler, MD, chose to pursue a fellowship at the School of Medicine because of the strength of its resources and expertise in NEC. She is a pediatric surgeon who will work in the lab of Brad Warner, MD, the Jessie L. Ternberg, MD, PhD, Distinguished Professor of Pediatric Surgery and the Children’s Hospital surgeon-in-chief, to explore small intestine regeneration. Her research seeks to understand the multi-level cellular interactions needed to grow the human epithelium that lines the intestine. That knowledge could advance approaches for organ regeneration.
At the University of Pittsburgh School of Medicine, Misty Good, MD, pediatrics, and her team used a mouse model to show that a compound in amniotic fluid inhibits gut inflammation. Now a neonatologist at Children’s, Dr. Good will use her CDI funding to explore the compounds in breast milk that offer the same protection and find out how to activate those compounds through nutritional supplementation.
With their CDI funding, Clifford Luke, PhD; and Stephen Pak, PhD, will capitalize on a worm model (C. elegans) of intestinal cells under attack to develop and perform drug screens for compounds that could prevent NEC. Identified targets then could be tested in mouse models.
These five studies and the ones that preceded them truly demonstrate the power of philanthropy to generate the kind of scientific inquiries that lead to deeper understanding of childhood diseases. Maybe one day, NEC will be something that can be accurately detected, treated and cured because, in part, of CDI donor funding.
Mary Dinauer, MD, PhD
Mary Dinauer, MD, PhD, is the scientific director of the Children’s Discovery Institute. She also is the Fred M. Saigh Distinguished Chair in Pediatric Research at St. Louis Children’s Hospital, professor of pediatrics and of pathology and immunology at Washington University School of Medicine.