Rapamycin and PKD (Polycystic kidney disease)

Hi everyone,

Polycystic kidney disease (PKD) is a genetic disorder that causes many fluid-filled cysts to grow in kidneys. Rapamycin has been looked at as a potential drug to target the disease. Actually, there was a clinical trial, back to 2006, looking into the usage of Rapamycin as a treatment for PKD, but the trail was ended with negative outcomes.

So, does anyone have an experience using Rapamycin to slow down the progress of the PKD disease?
Any comment is highly appreciated.


Hi and welcome to the forums. I’m not sure if anyone else here is familiar with PKD but hope they will respond if they are.

Do you know what the dosing protocol was in the failed clinical study? Do you have link or description of the study?

I can see the logic of rapamycin having some beneficial effects simply because it slows cell growth … but how that translates into benefits in this specific disorder is anyone’s guess.

Thanks for your reply @RapAdmin

There was a study (https://www.nejm.org/doi/full/10.1056/NEJMoa0907419) that looked into the subject. But based on the reported findings, there was no difference between the controlled group vs those who took Sirolimus.

I contacted Dr. Green, and he believed that rapamycin would be an excellent move to target PKD as the disease is proven to be an mTOR driven one.


Hello. Were you ever able to determine if rapamycin may be helpful per Dr. Green’s thoughts or further research? An acquaintance is fighting this disease. If you have further information it would be helpful for her. Thanks!