An international team of researchers including scientists from the Mayo Clinic and the University of Zurich have shown that tolvaptan, a drug already used to treat low blood sodium levels in patients with heart failure and liver problems, may be a promising new treatment for PKD. The findings are the culmination of 15 years of research.
In a recent three-year study of 1400 patients worldwide, tolvaptan reduced cyst growth and kidney growth, reduced pain, and slowed the decline in kidney function in patients with autosomal dominant polycystic kidney disease (ADPKD). The adverse effects of increased urination and thirst as well as increased liver enzymes and blood sodium levels did lead to a higher rate of discontinuation of the drug compared to patients taking a placebo.
Cystic Kidney Growth Curbed, ScienceDaily, November 5, 2012
Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease, New England Journal of Medicine, November 3, 2012