Testing at UT Southwestern has shown positive results for a new treatment of polycystic kidney disease, a genetic disorder that causes multiple cysts to form on the kidney and can lead to swelling and organ failure.
The study shows a 50 percent reduction in kidney size in mice following treatment. Dr. Vishal Patel, Associate Professor of Internal Medicine at UT Southwestern and senior author of the study has also led the drug to early clinical trials in humans.
Autosomal dominant polycystic kidney disease impacts around 12 million people worldwide, and about half end up with the final stages of kidney failure by age 60, where transplant or dialysis are the only options. A medicine called Jynarque is currently the only medicine approved to treat ADPKD, but side effects include “serious and potentially fatal liver injury.”
The new medicine was cooperatively developed by UT Southwestern and Regulus Therapeutics Inc., a biopharmaceutical company based in California, and showed no evidence of toxicity in animal of human cell tests.
“Once the kidneys have failed, the only options for survival are dialysis or a kidney transplant,” Patel said via release. “A large percentage of ADPKD patients on dialysis die each year while waiting for a donated kidney.”
A study describing the treatment and testing appears in this month’s Nature Communications.