Researchers have succeeded in developing a treatment against polycystic kidney disease which has shown encouraging results in animals.
A hereditary, incurable and often fatal genetic disease, polycystic kidney disease is characterized by the formation of cysts in the kidneys, which can lead to hypertrophy (an increase in volume) of both kidneys and, ultimately, kidney failure.
Of the approximately 12 million people affected worldwide, half would develop end-stage renal failure by the age of 60. “Once the kidneys fail, the only options for survival are dialysis or a kidney transplant,” says Vishal Patel, associate professor of internal medicine at UT Southwestern in Dallas, Texas.
Accompanied by his team, the latter nevertheless wants to be optimistic. He is the lead author of a study published this week in NatureCommunicationsin which he details the effects of a new treatment tested on mice with polycystic kidney disease: the size of their kidneys would have decreased by around 50%.
A drug with no signs of toxicity
Currently, only one drug has been approved by the US Food and Drug Administration (FDA) in the United States to treat polycystic kidney disease. Called Jynarque (generic name: tolvaptan), it can however cause “severe and potentially fatal liver damage”.
Developed with Regulus Therapeutics Inc, a California-based biopharmaceutical company, the new treatment developed by Professor Patel’s team showed no signs of toxicity in animals or in human cell trials, according to the study .
Another difference: it is preferably administered to the kidneys rather than the liver. “We have shown earlier that levels of a tiny fragment of ribonucleic acid (RNA) called microRNA-17 are increased in models of polycystic kidney disease,” explained Professor Patel. “This microRNA-17 interferes with the normal function of other beneficial RNAs, causing kidney cysts to grow. Currently called RGLS4326, the new drug in development works by blocking harmful microRNA-17.”
The first phase I clinical trials began last year, led by Regulus Therapeutics. The FDA has requested additional toxicity information from animal testing before human trials can proceed to the next stage. This new treatment is currently undergoing preliminary clinical trials in human subjects, pending FDA clearance.
.