Emerging therapies for polycystic kidney disease

Polycystic kidney diseases are the most common, monogenetic, inherited diseases in humans. Numerous human genes or gene loci are associated with a renal cystic phenotype. Currently, there are no treatments available to slow the development of renal cystic pathology; however, animal studies have identified several potential approaches to intervene in the disease process. The most advanced therapy is the use of vasopressin V2 receptor antagonists, which reduce renal cAMP, a known promoter of renal cystic enlargement. Other therapies under study include the use of c-myc antisense oligonucelotides and epidermal growth factor receptor tyrosine kinase inhibitors. Considering the diverse genes that cause renal cysts and the multiorgan involvement of these diseases, multiple therapeutic approaches will eventually be necessary to treat these diseases.