I did mention about the use of Rapamycin (see structure) to improve the efficacy of tuberculosis vaccine in my earlier blog. This drug has been already used as an immunosuppressant drug to prevent rejection in organ transplantation, especially useful in kidney transplants.
Rapamycin, was originally developed as an antifungal agent. However, this was abandoned when it was discovered that it had potent immunosuppressive and antiproliferative properties. Some researchers have also reported that the drug prolong the life of mice and might also be useful in the treatment of certain cancers.
Researchers from UC Santa Barbara earlier claimed that, rapamycin has a potential to treat kidney disease, however concluded that the mice had different genes affected than human patients. Interestingly, the same researchers recently found that "rapamycin is also highly effective in a new mouse model in which the same gene is affected as in most human patients".
As claimed by the lead researcher, Thomas Weimbs currently, no treatment exists to prevent or slow cyst formation and most ADPKD patients require kidney transplants or lifelong dialysis for survival. I think this will boost the confidence of the several international groups, who are undertaking the clinical trials to test the safety and efficacy of rapamycin and related drugs in polycystic kidney disease. Though the researchers are hopeful of positive results they caution that, it will be critical to balance any benefits against the expected side effects to judge whether these drugs should be recommended for the treatment of polycystic kidney disease. Let us be optimistic.....
Ref : http://www.ia.ucsb.edu/pa/display.aspx?pkey=2164