Incurable, deadly kidney disease sees promise with new US drug
Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder where multiple cysts form on the kidneys and quickly leads to painful swelling and end-stage kidney disease or organ failure. A recent study by the University of Texas Southwestern Medical Center (UTSW) highlighted a potential treatment for the incurable ADPKD with a drug that showed nearly a 50% reduction in kidney size (swelling) in a laboratory experiment.
While the current drug to treat ADPKD is likely to cause liver damage, the new treatment is delivered to the kidneys rather than the liver after being administered to patients. It also showed no obvious toxicity in animals or in human cells after stringent testing.
Dr. Vishal Patel, Associate Professor of Internal Medicine at UTSW, laments that a large number of ADPKD patients on dialysis die each year while waiting for a donated kidney.
According to Dr. Patel, “The levels of a tiny RNA fragment called microRNA-17 are increased in models of ADPKD – it interferes with the normal function of other, beneficial RNAs, causing kidney cysts to grow. The new drug works by blocking the harmful microRNA-17.”
Early phase one clinical trials began last year, but additional toxicity information from animal testing is required before human trials can continue, Dr. Patel said.
