Across numerous medical specialties, physician scientists and researchers at Riley Children’s Health are making significant strides in pediatric research funded by the National Institutes of Health (NIH) and other major agencies. In federal fiscal year 2023, Riley Children’s and the Indiana University School of Medicine Department of Pediatrics ranks #9 in NIH-funded pediatric research, amassing over $38.5 million in grants for numerous studies. Here are some notable highlights:
Research reveals insights for safer hemophilia treatments
With a $12 million grant from the National Heart, Lung and Blood Institute (NHLBI), researcher and Professor of Immunology at the Riley Children’s Foundation, Roland Herzog, PhD, leads a collaborative study spanning multiple institutes. The project explores a gene therapy approach targeting safer and potentially curative treatments for hemophilia A. The basic and translational studies explore cellular toxicity and stress induced by FVIII protein production, molecular virology and the immune system’s role in interfering with FVIII production, ultimately creating a better gene therapy protocol.
Solutions for personalized therapies for severe asthma
Pediatric pulmonologist at Riley Children’s, Benjamin Gaston, MD, is leading a five-year, $12 million program project grant (P01) from the NHLBI. The grant, Scientific Innovation for Personalized Severe Asthma Management, funds the development of personalized therapeutic approaches, exploring three topics related to S-nitrosylation, airway pH regulation and androgen signaling. Having studied these mechanisms for more than a decade, the research group, which includes investigators from Case Western Reserve University, found that they 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. To learn more, view the latest pulmonology brochure.
National research effort targets treatments for inherited childhood cancers
D. Wade Clapp, MD, chairman of pediatrics at Riley Children’s, spearheads a collaborative research study funded by a $11.4 million Specialized Programs of Research Excellence (SPORE) grant from the National Cancer Institute (NCI). He, alongside national collaborators, strives to develop new treatments for tumors associated with neurofibromatosis type 1 (NF1), the most common inherited syndrome predisposing individuals to cancer. Furthermore, the SPORE grant, with a strong focus on childhood cancer, studies the abnormal activity of Ras, a key signaling protein involved in over a third of all cancers, potentially leading to breakthrough therapies for NF1, as well as breast and lung cancers, pediatric and adult brain tumors, melanoma and acute myeloid leukemia. Presently, this SPORE grant is the only active pediatric-focused project.
Novel blood pump offers hope for patients with single ventricle heart disease
Mark D. Rodefeld, MD, pediatric cardiothoracic surgeon at Riley Children’s, secures a $9 million NIH grant for his innovative work on a blood pump designed to provide cavopulmonary assist in a univentricular Fontan circulation. The novel implantable pump aims to permanently reverse the univentricular Fontan circulation, halting the progression of chronic Fontan disease. As a result of the study, the device will significantly improve the overall quality of life and outcomes for children and adults born with single ventricle heart disease.
Study aims to cure congenital heart defects in patients
Led by IU School of Medicine researcher Anthony B. Firulli, PhD, the program project grant (P01) funded by the NHLBI aims to understand the molecular mechanisms that drive ventricular morphogenesis. Aiming to minimize congenital heart defects in infants, the project encompasses three comprehensive studies. The first study examines the molecular mechanisms contributing to the genesis of CHDs in an animal model of X-linked heterotaxy. The second investigates molecular mechanisms regulating ventricular septation and papillary muscle formation. The third project analyzes the molecular mechanisms regulating compaction of the left ventricle during cardiac development.
Overall, Riley Children’s, in conjunction with the Herman B Wells Center for Pediatric Research at IU School of Medicine, is a leader in pediatric research, translating discoveries into innovative solutions and life-altering breakthroughs for children’s health. To learn more, read the annual report.
