The pathophysiology of ADPKD is complex and various potential therapeutic targets are available for which novel treatments are being developed or repurposed, but as yet V2RAs are currently the only widely accepted renoprotective treatment for this disease.

Because ADPKD is a chronic disease that requires lifelong treatment, and targets involved in cyst growth also have important functions in various physiological processes, there is a need to develop (targeted) therapies to minimize systemic side effects.

Since various treatment options have shown discrepant results between preclinical and clinical studies, we suggest the use of at least 2 different rodent polycystic kidney disease models before proceeding to clinical trials or before aborting drugs for clinical use.