IU researchers launch human clinical trial to halt progression of polycystic kidney disease

September 24, 2015

A study on the treatment of polycystic kidney disease, initially funded by the Indiana Clinical and Translational Sciences Institute, has gained new momentum and is now entering the clinical trial phase under the guidance of a team of Indiana University researchers and a colleague at the Mayo Clinic.

Treatment for PKD to enter clinical trial phase | Photo By Bonnie Blazer-Yost

Polycystic kidney disease affects more than 1 in 1,000 Americans, causing cysts to form on the kidneys. The cysts cause the kidneys to enlarge, sometimes becoming as big as footballs, leading eventually to kidney failure.

Under a three-year, $600,000 Food and Drug Administration grant, Bonnie Blazer-Yost, Ph.D., and Sharon Moe, M.D., will serve as co-principal investigators into whether pioglitazone, also known as Actos, is an effective long-term therapy to stop autosomal dominant polycystic kidney disease.

While healthy adult kidneys are the size of a fist and weigh less than a pound, polycystic kidneys can weigh as much as 20 to 30 pounds. The fluid-filled cysts initially grow out of nephrons, tiny filtering units within the kidney, but eventually separate from the nephrons and continue their growth while the kidney enlarges as well.

“At present, there are no FDA-approved drugs to treat PKD and the only therapies are to either aspirate the larger cysts to relieve the pain or perform a transplant once the kidney fails,” Blazer-Yost said,  “What we hope to demonstrate is the ability to halt or greatly inhibit cystic growth in our trial participants. If this proves successful, it will lead to larger trials with the ultimate goal of having a treatment for PKD that can be used as a lifelong medication.”

 Blazer-Yost is a biology professor at Indiana University-Purdue University Indianapolis, while Moe serves as a professor and chief of nephrology at the Indiana University School of Medicine. Robert Bacallao, M.D., an associate professor of medicine and director of the Polycystic Kidney Disease Clinic at the IU School of Medicine, also will serve as a co-investigator. Vicente Torres, M.D., Ph.D., a professor of medicine at the Mayo Clinic’s Division of Nephrology and Hypertension in Rochester, Minn., will consult and provide magnetic resonance imaging interpretation for the study.

 To learn about this research, visit the IU Newsroom.