Author: ["Vincent H Gattone II","Xiaofang Wang","Peter C Harris","Vicente E Torres"]
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Abstract
The polycystic kidney diseases (PKDs) are a group of genetic disorders causing significant renal failure and death in children and adults. There are no effective treatments. Two childhood forms, autosomal recessive PKD (ARPKD) and nephronophthisis (NPH), are characterized by collecting-duct cysts1,2. We used animal models orthologous to the human disorders to test whether a vasopressin V2 receptor (VPV2R) antagonist, OPC31260, would be effective against early or established disease. Adenosine-3′,5′-cyclic monophosphate (cAMP) has a major role in cystogenesis3,4, and the VPV2R is the major cAMP agonist in the collecting duct5,6. OPC31260 administration lowered renal cAMP, inhibited disease development and either halted progression or caused regression of established disease. These results indicate that OPC31260 may be an effective treatment for these disorders and that clinical trials should be considered.Cite this article
Gattone, V., Wang, X., Harris, P. et al. Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist. Nat Med 9, 1323–1326 (2003). https://doi.org/10.1038/nm935 