Treatment for polycystic kidney disease shows promising results

A potential treatment for polycystic kidney disease, which causes the kidneys to swell with multiple cysts, has shown promising results in animal testing, with approximately 50 percent reduction in kidney size in afflicted mice following treatment.

Polycystic kidney disease causes the kidneys to swell with multiple cysts. The new treatment showed a reduction of 50 percent kidney size in animal testing (credit: UT Southwestern).

The new treatment for autosomal dominant polycystic kidney disease (ADPDK) cooperatively developed at UT Southwestern, US and Regulus Therapeutics Inc, US showed no evidence of toxicity in animals or in human cell tests, according to the study. It is preferentially delivered to kidneys rather than the liver after being administered.

“We earlier showed that levels of a tiny RNA fragment called microRNA-17 are increased in models of ADPKD,” said Dr Vishal Patel, Associate Professor of Internal Medicine at UT Southwestern and senior author of the study. “MicroRNA-17 interferes with the normal function of other, beneficial RNAs, causing kidney cysts to grow. RGLS4326, as the new drug is called in development, works by blocking the harmful microRNA-17.”

Early Phase I clinical trials for the new treatment for polycystic kidney disease began last year. The US Food and Drug Administration (FDA) has asked for additional toxicity information from animal testing before human trials can move to the next step, Dr Patel said.

The study was published in Nature Communications.