Is Metformin Now in Our Armamentarium for Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

Treatment options for autosomal dominant polycystic kidney disease (ADPKD) are sparse despite the detrimental course of the disease. Tolvaptan, a vasopressin V2 receptor (V2R) antagonist, is the only FDA approved treatment so far to decelerate decline in glomerular filtration rate (GFR) in ADPKD. There has been a pressing need for additional therapeutic strategies that can prevent kidney enlargement and progression to end-stage kidney disease (ESKD), which occurs in almost half of the patients with ADPKD. In that regard, metformin has been investigated in preclinical and clinical studies with a proposed mechanism of AMP-activated protein kinase (AMPK) activation.

For the past 20 years, researchers have used rodent models of ADPKD to investigate efficacious therapies for ADPKD. Read the full article in the American Journal of Kidney Diseases blog.