Table 3.

Select novel therapies in PKD

Drug name/class	Mechanism of action	Supporting data
mTOR inhibitors	Inhibition of mTOR signaling cascade, disrupting renal epithelial cell proliferation	Randomized, single-blind study showing significant slowing of renal size in patients treated with rapamycin and telmisartan, compared to telmisartan alone
Vasopressin receptor antagonists	Disruption of binding of vasopressin to V2 receptors, inhibiting cAMP-associated signaling cascades	Phase 2 results show tolvaptan to be safe and well-tolerated; phase 3 studies in progress
Octreotide	Inhibition of cAMP production	Shown in a randomized, cross-over, placebo-controlled trial to significantly slow renal volume expansion; no effect on GFR

Abbreviations: mTOR, mammalian target of rapamycin; cAMP, cyclic adenosine monophosphate.