Across multiple specialties, physician scientists and researchers at Riley Children’s Health are taking aim at complex pediatric diseases with groundbreaking research funded by the National Institutes of Health and other major funding agencies. In federal fiscal year 2022, the Indiana University School of Medicine Department of Pediatrics and Riley Children’s are ranked 7th in NIH-funded pediatric research, receiving more than $38 million in NIH grants for 76 studies. Here are some highlights:
Research underway to find safer hemophilia treatments
With a $12 million grant from the National Heart, Lung, and Blood Institute, Riley researcher Roland Herzog, PhD, is leading a multi-institute effort exploring three major themes in a gene therapy approach that could lead to safer—and potentially curative— treatments for hemophilia A. The basic and translational studies focus on 1) cellular toxicity and stress that can be induced by FVIII protein production (patients with hemophilia A do not produce sufficient amounts of this clotting protein); 2) molecular virology and the development of viral vectors used in gene therapy to deliver the FVIII encoding gene; and 3) the immune system and its role in the interference of FVIII production over time.
With P01 grant, team develops personalized therapies for severe asthma
A research team led by Riley pulmonologist Benjamin Gaston, MD, is using a P01 program grant from the National Heart, Lung, and Blood Institute to fund the development of personalized therapeutic approaches for severe asthma. Riley Children’s is one of the only pediatric pulmonology programs in the nation with a P01. For more than a decade, Dr. Gaston’s research group has studied S-nitrosylation signaling, airway pH regulation and androgen signaling and has shown that these mechanisms not only indicate the type of severe asthma a person has, but are also potential targets for treatment. By the end of the research program grant, the group anticipates developing at least three novel approaches to managing severe asthma. Overall, Riley Pulmonology is engaged in nearly 140 IRB-approved clinical research projects and 15 basic science projects.
National work focused on treatments for inherited childhood cancers
Fueled by a $11.4 million Specialized Programs of Research Excellence (SPORE) grant from the National Cancer Institute, D. Wade Clapp, MD, physician-in-chief at Riley Children’s, is leading a collaborative research effort to develop new treatments for tumors that develop in neurofibromatosis type 1 (NF1). The most common inherited syndrome causing a predisposition to cancer, NF1 affects one in every 3,000 people worldwide, with about 100,000 individuals living with the syndrome in the United States.
With a strong childhood cancer focus, the SPORE grant is a multi-institutional effort studying the abnormal activity of Ras, a key signaling protein, which is involved in more than a third of all cancers. The research could lead to new therapies for NF1 and other cancers, including breast and lung cancers, pediatric and adult brain tumors, melanoma and acute myeloid leukemia.
Revolutionary blood pump improves long-term outcomes for single ventricle heart disease
Riley cardiothoracic surgeon Mark D. Rodefeld, MD, has received multiple National Institutes of Health grants for his work to develop a blood pump designed to provide cavopulmonary assist in a univentricular Fontan circulation. Those who survive Fontan repair often have chronic circulatory issues and carry a lifelong risk of failure with survival rates 30 years after Fontan surgery of 43 to 70%. By permanently reversing the univentricular Fontan circulation, Dr. Rodefeld’s implantable pump halts the progression of chronic Fontan disease, significantly improving quality of life and outcomes for children and adults born with single ventricle heart disease.
The ability to bring new discoveries to patient care sets Riley Children’s apart from other pediatric healthcare providers in the Midwest. In partnership with Riley Children’s, the Herman B Wells Center for Pediatric Research at IU School of Medicine works to find new technologies and treatment options for children and is nationally recognized for life-sustaining innovations.