As we reflect on 2025, we celebrate the extraordinary progress we’ve made together in advancing child health research. Read on for impactful stories from our partners highlighting recent discoveries by our Department of Pediatrics faculty.
CU physician David Fleischer, MD, explains why the new technology, which is moving toward FDA approval, is a positive step for early intervention and how it may alleviate food allergy concerns for families.
Christopher Stille, MD, founded and continues to co-lead a national research network to support health systems for children with special health care needs and their families.
Led by principal investigator Kevin Messacar, MD, and first author Hai Nguyen-Tran, MD, the study highlights how real-time surveillance in children can enhance disease modeling and improve public health preparedness for future outbreaks.
One in a million.
Those are the odds of having C3 glomerulopathy (C3G) or immune complex membranoproliferative glomerulonephritis (IC-MPGN). For decades, there has been no targeted treatment for these rare and severe kidney diseases. Bradley Dixon, MD, wanted to change that. And he did.
Dr. Dixon, a pediatric nephrologist, is a co-author on the recently published VALIANT study in The New England Journal of Medicine. The data and research validated through this study paved the way for the Food and Drug Administration’s (FDA) approval of pegcetacoplan as a treatment for individuals 12 and older.
Years out from treatment for cancer, some children may get secondary treatment-induced high-grade glioma. Specifically, children who are treated with cranial radiotherapy can develop incurable secondary brain tumors called pediatric radiation-induced gliomas. As a neuro-oncologist, Adam Green, MD, witnessed the impact of these brain tumors and saw the need for research that could give children a second chance at life.
“The CFTR modulator is a medication that helps the CF protein work better in people with CF,” said Jordana Hoppe, MD, a pediatric pulmonologist at Children’s Hospital Colorado. She has been one of the pulmonologists caring for the Kent children since they were infants and was a lead principal investigator for the pediatric ALYFTREK trials. The Colorado Clinical and Translational Sciences Institute (CCTSI) provided the expert research nurses and the pediatric Clinical Translational Research Center where this CF trial took place. Indeed, all CF trials over the past 15 years have relied on these CCTSI resources and services.
“These are very complex new treatments that are coming online, and they're different than traditional drug trials and pharmaceuticals that are coming to market,” says David Brumbaugh, MD, Chief Medical Officer at Children’s Hospital Colorado.
Coordinating delivery of these complex novel therapeutics is no small feat, according to ... Meg Macy, MD, Medical Director of the Colorado Child Health Research Institute.
“There's a tidal wave coming of these types of treatments,” Scott Demarest, MD, Chief Precision Medicine Officer says. “These are new therapies for us organizationally, and we want to be the go-to place because we have built the infrastructure to deliver them efficiently, in cost-effective manners, sustainably, and above all else, safely for patients.”
Experts at Children’s Hospital Colorado, including Kelly Wolfe, PhD, Jesse Davidson, MD, MPH, MSc, Sherrill Caprarola, MD, MSHS, and Sarah Kelly, PsyD, have reported new evidence suggesting that young children with congenital heart disease (CHD) who receive neurodevelopmental care while hospitalized for longer periods (a week or more) may experience a lower incidence of delirium and better cognitive outcomes.
According to Kristen Nadeau, MD, MS, pediatric endocrinologist and principal investigator of RISE and DISCOVERY, the TODAY study’s (chaired by Phil Zeitler, MD, PhD) findings were stunning: Most teens lost blood sugar control rapidly, regardless of treatment. Even combining two medications didn’t guarantee success, and lifestyle interventions weren’t enough. A central takeaway from this research was that compared to adults, youth had higher failure rates and increased resistance to insulin.
TODAY tracked long-term outcomes as patients aged into their 20s and 30s — and what they found was perhaps even more alarming. As they aged, patients with youth-onset T2D began to experience serious complications far earlier than expected: heart attacks, strokes, kidney failure and other life-altering events that typically don’t occur until much later in life. “These complications are occurring at a time when you’d expect people to be the healthiest in their lives and possibly reproducing,” says Megan Kelsey, MD, MS, pediatric endocrinologist and site principal investigator for DISCOVERY.
Jamie Feinstein, MD, MPH, professor of pediatrics, who appears in the new film focused on finding a cure for epidermolysis bullosa, says global awareness is a critical piece of stopping the devastating genetic skin disease.
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