The National Institutes of Health (NIH) and the PKD Foundation have launched two treatment trials for autosomal dominant polycystic kidney disease (ADPKD).
The common inherited disorder is characterized by cysts in the kidneys and other organs, high blood pressure, and aneurysms (bulges in blood vessels, which may burst) in the brain. Symptoms usually appear between the ages of 30 and 40 and include back and side pain and headaches. About half of ADPKD patients eventually develop kidney failure and require dialysis or a kidney transplant. The first Halt Progression of Polycystic Kidney Disease (HALT-PKD) patient enrolled last week at Emory University in Atlanta, one of seven recruitment sites .
"Decades of clinical and basic studies by NIH and others have delivered this exciting opportunity for translational research," says Catherine M. Meyers, M.D., a kidney specialist who directs HALT-PKD at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). "Naturally, we appreciate the PKD Foundation's invaluable guidance and support."
PKD Foundation President and CEO Dan Larson applauded the start of the trials. "PKD families are eager to learn of any potential benefits," says Larson. "Their hope and the hope of the PKD Foundation is that this will be a step toward finding a cure for PKD and improving the care and treatment of those it affects."
Carefully controlling blood pressure and using ACE-inhibitors or ARBs significantly delays or prevents kidney disease and failure from diabetes and other causes by reducing protein in the urine and preventing damage to the small blood vessels in the kidneys. Earlier trials of these treatments in PKD were not definitive, possibly because a small number of patients were involved.