| Triptolide |
Stimulates polycystin 2-dependent calcium release |
|
Kidney specific Pkd 1-/- mouse |
|
Improved renal function |
| CFTR Inhibitors |
Interferes with fluid secretion |
|
Kidney specific Pkd 1-/- mouse |
|
Improved renal function |
| Octreotide |
Inhibits cAMP |
|
PCK rat |
|
Decreased the formation of cysts in the liver and kidney but no improvement in renal function. |
|
Randomized crossover placebo-controlled |
Smaller increase in kidney volume |
| Vasopressin antagonists |
Lowers cAMP |
pcy mouse |
PCK rat Pkd2 (ws25/-) mouse |
|
Lowered kidney weights,Cr, BUN |
| PCK and Brattleboro rat |
|
PCK rats that lacked vasopressin show reduced cyst formation |
| ongoing |
TEMPO, a phase III study is underway |
| Pioglitazone |
Possibly inhibits β-catenin signaling |
|
Pkd1-/- mouse |
|
Improved phenotype and molecular defects. |
| Roscovitine |
Inhibits cyclin dependent kinases |
jck mouse cpk mouse |
|
|
Intermittent administration of roscovitine showed a long lasting benefit |
| MAPK/ERK inhibitor |
Inhibits MAPK/ERK signaling |
pcy mouse |
Kidney-specific Pkd1-/- mouse |
|
Decreased cystic index in the pcy mouse but no beneficial effect in the kidney-specific Pkd1 mutant mouse |
| Src Inhibition |
Decreases ErbB1 and ErbB2 activity |
bpk mouse |
PCK rat |
|
Src inhibition ameliorated renal and biliary lesions |
| mTOR inhibitors |
Prevents mTOR mediated cell growth |
Han:SPRD rat oprk-rescue mutant mouse, bpk mouse |
|
Retrospective studies |
Decreased kidney volume in one study. Second study showed decrease in liver volume but not in kidney volume. |
| Etanercept |
Inhibits TNF-α |
|
Pkd2+/- mouse |
|
TNF-α stimulated and etanercept inhibited cyst formation. |
