December 12, 2014
By Andy Hyland
|Jared Grantham, M.D.|
A large clinical trial has demonstrated for the first time the benefits of rigorously controlling high blood pressure for patients with polycystic kidney disease (PKD). Researchers at the University of Kansas Medical Center are members of a national team that have published the results of new landmark studies for patients with the condition.
Jared Grantham, M.D., University Distinguished Professor Emeritus and Director Emeritus of the KU Kidney Institute, and Franz Winklhofer, M.D., are co-authors of two papers outlining the results of the trial that have been published online in the New England Journal of Medicine. Lead authors on the papers were Arlene Chapman, M.D., of the Emory University School of Medicine; and Vicente Torres, M.D., Ph.D., of the Mayo Clinic.
PKD is a chronic and often painful condition that can be life-threatening. Patients with PKD develop fluid-filled cysts in their kidneys that cause the kidney to enlarge, leading to kidney failure. The condition is genetic, and parents have a 50 percent chance of passing the disease on to each of their children. There is no known cure for PKD.
The disease is one of the most common of all life-threatening genetic diseases, affecting hundreds of thousands of people in the United States and millions worldwide.
By closely controlling patients' blood pressure, the research demonstrates that the growth of the kidneys can be slowed and that injury to the heart and blood vessels can be eased, possibly reducing harm to kidney function over the long term. Grantham said the researchers studied 1,044 patients in sites across the country, and 83 people participated in the study at KU Medical Center. Researchers followed patients for up to eight years during the duration of the study, which concluded in June 2014.
"While researchers have long suspected that paying very close attention to blood pressure early on will benefit these patients, this is the first large study to really nail that down with good, hard scientific data," Grantham said. "This is an important step as we move closer to a cure for PKD."
Grantham, an internationally known kidney expert, and his colleagues at KU Medical Center have a long history of success in PKD research dating back to the 1970s. Grantham said this new knowledge builds on other major milestones in the fight against PKD, including a recent clinical trial that showed that a drug developed as part of KU Medical Center research could also slow the progression of the disease.
Winklhofer treated participants in the trial at KU Medical Center, who were seen in facilities provided by Frontiers: The Heartland Institute for Clinical and Translational Research.
Anne Bartels, a PKD patient from Lee's Summit, Mo., enrolled in the trial at KU Medical Center. Her grandmother and her mother both had PKD as well, and she was diagnosed at age 32.
"That's why I'm very involved in the study," she said. "To help find a cure for future generations. I got involved right after I was diagnosed. It's a not a disease that's talked about very often, so I try to raise awareness for it."
Grantham also co-founded the PKD Foundation, a national organization headquartered in Kansas City, Mo. It is dedicated to discovering treatments and a cure for PKD and improving the lives of all it affects.
The foundation's leaders said the foundation was proud to have funded and supported this work, along with the National Institute of Diabetes and Digestive and Kidney Diseases, and they were happy to see the positive outcome.
"We're so pleased to learn about the results of this milestone study. The information gained from these trials is invaluable in understanding PKD, and identifying potential ways to slow its progression," said Jackie Hancock, Jr., CEO of the PKD Foundation. "This is important progress that will keep moving us closer to ending PKD."
This work was supported in part by grants from the NIDDK (#DK62402, #DK62411, #DK62410, #DK082230, #DK62408 and #DK62401) and the National Center for Advancing Translational Sciences through Frontiers (#TR000001).