Pilot & Feasibility Program (PFP)

The Pilot and Feasibility program encourages young and established investigators to undertake research relevant to kidney development, engineering and disease multiomics to accelerate the acquisition of knowledge that can be applied to treatment of children with renal and urological diseases and engineer devices to replenish kidney function.

Awardees

PI	Award year	Career stage	Institution	Title:	Description:
Kyle McCracken, MD, PhD	2023	Early	Cincinnati Children’s Hospital Medical Center	Regulation of cell fate determination in the ureteric bud lineage in hPSC-derived organoids	In this project, we will use human pluripotent stem cell-derived organoids and fluorescent lineage reporters to elucidate the mechanisms that regulate cell fate decisions in the developing urinary collecting system. These pilot studies will inform our approach and ability to generate distinct types of epithelial tissues from stem cells, including the ureter and collecting duct, which will have diverse applications in kidney tissue engineering and disease modeling.
Moe R. Mahjoub, PhD	2023	Established	Washington University in St. Louis	Characterizing Centrosomal Pathways Underlying Congenital Kidney Developmental Defects and Early-Onset Fibrocystic Disease	Congenital fibrocystic kidney “ciliopathies” comprise a group of pediatric disorders caused by mutations in genes associated with the centrosome-cilium organelle complex. There are no effective molecular therapies that successfully halt disease progression. Understanding the underlying cellular and molecular mechanisms that contribute to the disease is critical in developing an effective therapeutic strategy. The goal of this project is to identify pathways that are disrupted in the rare, early-onset pediatric renal ciliopathy diseases caused by mutations in centrosome-associated genes. These experiments will be performed using a new mouse model of pediatric ciliopathies, together with single-cell sequencing approaches.
Albert Liu, MD	2023	Early	Washington University in St. Louis	Mechanism of Hypertension in Metabolic Syndrome and Post-Transplant Obesity and Diabetes	Metabolic syndrome is a complex constellation of obesity, insulin resistance, dyslipidemia, and hypertension. In metabolic syndrome, the hypertension is thought to be due to endothelial dysfunction as a result of the other components of metabolic syndrome. Glucagon-like peptide-1 receptor agonists (GLP-1RA) are a class of medication originally approved for type 2 diabetes mellitus with recent approval for obesity, and use of these medications have been colloquially associated with decreased need for antihypertensive medications. We aim to assess the relationship of GLP-1RA use and associated need for antihypertensive medications, and also assess vascular function using non-invasive methods (pulse wave analysis, pulse wave velocity, carotid intima-media thickness, and flow-mediated dilation) to assess vascular functioning in pediatric patients with metabolic syndrome as compared to healthy individuals, as well as assess changes in these assessments in patients with metabolic syndrome receiving GLP-1RA therapy.