Shedding Light on Rare Kidney Diseases

The Division of Nephrology’s Sharon Bartosh, MD, along with her colleagues, is part of a national, multi-site, National Institutes of Health-sponsored trial that aims to understand more fully and improve treatment for a set of kidney diseases known to affect children and adults. 

The four conditions being studied damage the glomeruli, the part of the kidney that keeps cells and protein in the blood while filtering waste, salts and leftover fluid into the urine.

Without proper treatment, people with glomerular disease are at high risk for developing kidney failure, and may require long-term dialysis or transplant.

Elucidating Causes, Progression and Therapy

The trial, CureGN, is a five-year study that aims to enroll 2,400 children and adults at over 50 centers in the U.S., Canada and Italy.

It focuses on four specific types of glomerular disease: minimal change disease, focal segmental glomerulosclerosis (FSGS), membranous nephropathy and IgA nephropathy/HSP (Henoch Schonlein Pupuric) nephritis.

By following patients longitudinally, study investigators aim to better understand the genetic and biological causes of the diseases, their progression and how patients respond to therapy—with cure being the ultimate objective.

The trial is funded by the National Institutes of Health–National Institute of Diabetes and Digestive and Kidney Diseases.

Improving Clinical Knowledge Nationwide

Dr. Bartosh is participating in the trial through the Midwest Pediatric Nephrology Consortium (MWPNC), a group of pediatric kidney centers who work together to find the best treatments for patients. Of the more than 50 MWPNC members, 32 are participating in the CureGN trial.

The other CureGN centers are Columbia University, the University of North Carolina and the University of Pennsylvania.

Since June 2015, Dr. Bartosh’s team has enrolled 16 patients age 4 through 19 in the trial. Participating patients have all four types of glomerular disease; some are in the earliest stages of their disease, while others have more advanced disease requiring multiple medications.

Throughout the study, patients provide blood and urine samples, which are analyzed for biomarkers of disease progression. Study coordinators also stay connected with patients via telephone to track important clinical events related to their disease.

Over time, the trial will improve clinical knowledge about how to best treat glomerular disease. Study coordinator Barbara Bowman noted that because previous research on the diseases has been scant, treatment is often inconsistent from center to center.

“Given that kidney disease is rare in children, it is essential that pediatric nephrologists and our patients participate in endeavors such as this trial,” Dr. Bartosh noted.

“It is unusual for any one center to have enough pediatric kidney patients to allow rigorous academic study of them and their responses to therapy,” she continued. “The CureGN opportunity allows us to pool our patient data and responses to therapy in such a way that will allow us to gain insights into genetic associations, and determine which therapeutic strategies are most effective and for which patient populations.” 

For more information about study enrollment, contact Barbara Bowman at (608) 265-2645 or babowman@pediatrics.wisc.edu