Pioglitazone, may halt the growth of cysts in Polycystic Kidney Disease....

Recently, researchers from the schools of Science and Medicine at Indiana University-Purdue University Indianapolis and colleagues from the Mayo Clinic reported that drug Pioglitazone, (see structure Enantiomers) commonly used to treat diabetes may also retard the growth of fluid-filled cysts of the most common genetic disorder, polycystic kidney disease.

Using a rat model that has the same genetic mutation as a form of human PKD, the two research groups independently tested a pioglitazone treatment regimen and found that it slowed down both kidney and liver cyst growth by inhibiting a chloride channel in the cells of these organs. Authors claim that the, though the idea of using a chloride channel inhibitor to treat PKD is not new, but usage of an insulin sensitizing agent like piogltiazone inhibits chloride channels is new. The finding that pioglitazone, which has already been approved by the Food and Drug Administration for diabetes, can halt cyst progression and may be an effective and well-tolerated treatment for this chronic disease, is exciting. Confirmation of these results in other animal models of PKD would be a useful next step.....

Ref : http://medicine.iupui.edu/GAST/NEJM%20Article.pdf

Rapamycin as a potential treatment for kidney disease (ADPKD).........

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