Despite being a frequent cause of end-stage renal disease (ESRD), there are a lack of treatment options to manage autosomal-dominant polycystic kidney disease (ADPKD) and slow down the loss of kidney function. A study published in the Clinical Kidney Journal looks into drugs that have been repurposed and successfully used in clinical trials for ADPKD treatment and how they could improve management of this disease.*

Background information

ADPKD is a severe kidney condition associated with kidney enlargement; the formation of cysts in the kidney and other organs; flank (upper abdomen or back and sides) pain; and progressive loss of kidney function.

In the past, the only therapeutic options available to alleviate ADPKD symptoms were supportive procedures used for other chronic kidney diseases (CKD).

These supportive measures included:

• blood pressure control;
• limiting salt intake (less than 5–7 gram/day);
• sufficient fluid intake (more than 3 liters per day);
• a healthy diet; 
• increased physical activity; and
• controlling one’s body weight.

What they did

In this study, researchers from the University of Cologne in Germany discussed the current management of ADPKD patients and the interventions used to maintain kidney function.

The study considered both:

• current and future clinical trials, and 
• potential novel ADPKD treatment strategies. 

Recent interventional trials for finding ADPKD treatment options included the following medications: mTor inhibitors, Tolvaptan, somatostatin analogs, and tyrosine kinase inhibitors.

What they found

The use of drugs with long-standing clinical experience for other conditions and a good safety profile was an attractive strategy for repurposing treatments for ADPKD. 

A combination of the following show promise to improve the treatment of ADPKD:

• supportive procedures,
• identification of new disease-modifying drugs, and
• repurposing of drugs approved for other diseases.

Statins are good examples of such an approach. Lovastatin, for example, has shown to be beneficial in preserving kidney function and preventing cyst growth in the rat model. A positive effect of another statin, pravastatin, was confirmed in a pediatric Phase 3 clinical trial that examined its effect (versus a placebo) in 110 children.

What it means

A number of big clinical trials indicate that a mixture of managing blood pressure, body weight, and diet—when combined with disease-modifying drugs—may be the key for ADPKD patients. Repurposing drugs and combining different therapies may improve the pharmacological treatment of ADPKD while “limiting side effects to a tolerable level.”

More clinical trials will be necessary, however, to further understand how to best manage this disease.

*Müller, R-U., & Benzing, T. (2018, Dec. 1). “Management of autosomal-dominant polycystic kidney disease—state-of-the-art.” Clinical Kidney Journal.