Polycystic kidney disease future or investigational therapies

• It is known that a certain molecule known as cAMP is involved in the enlargement of kidney cysts in PKD kidneys.^[1]
• Various studies on rodents have shown that a molecule, called vasopressin, increases levels of cAMP in the body. When mice with PKD were given a chemical that blocks vasopressin, there was an impressive decrease in kidney size and some preservation of kidney function.
• Similarly, when studied mice consumed excessive amounts of water (which decreases levels of vasopressin), a similar result was seen. It has therefore been suggested that consuming large amounts of water may possibly assist in the treatment of early stage PKD.^[2][3]
• As humans do not always mimic rodents in clinical trials, it is currently not yet certain whether vasopressin inhibitors, such as water, will have corresponding results in humans, or what negative effects excessive water intake may have on the kidneys of individuals with PKD.
• Clinical trials are currently underway in this field.^[4]
• It has also been suggested that treatment with medications inhibiting vasopressin may assist in the management of PKD and reduce the speed at which kidney cysts form and grow, delaying the onset of end stage renal failure.
• Clinical trials are currently underway in the testing of Tolvaptan, a U.S. Food and Drug Administration (FDA) approved medication which has not yet been approved for use in the treatment of PKD.^[5]

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