Clinical Trial Planned for Rapamcyin

Plans are now underway for two clinical trials to look at how rapamycin could reduce the size of enlarged kidneys in patients with PKD.

The first study, which will take place in Switzerland, is already enrolling patients. The 24 month study at the University of Zurich will investigate whether rapamycin retards cyst growth, therfore, slowing the decreased kidney function in patients with PKD. Renal volume will be measured by magnetic resonance imaging (MRI). More information about the study and eligibility criteria can be found by clicking here or by contacting Dr. Andreas Serra, Principal Investigator, at  andreas.serra@usz.ch

The second clinical trial, sponsored by the Cleveland Clinic, is still in the planning phases and is not yet open for patient recruitment. For more information, click here, type in “polycystic kidney disease” and then select “Pilot Study of Rapamycin as Treatment for Autosomal Dominant PKD.” Anyone interested in the clinical trial can also contact Susan Wirth, BSN, at wirths@ccf.org

What is rapamycin? Rapamycin, also called sirolimus, is an immunosuppressant. The drug is prescribed to help prevent rejection of a new, transplanted kidney. Researchers led by Dr. Weimbs at the University of California-Santa Barbara tested rapamycin in mice and also in a small group of transplant patients. They found the patients’ polycystic kidneys shrank by 25 percent over two years. According to an article in the Los Angeles Times, the drug has already been approved by the Food and Drug Administration and is expected to lead to additional clinical trials soon.

Want to learn more? Click here for a 20-minute presentation on rapamycin from researcher Dr. Thomas Weimbs. To read the full Los Angeles Times article, click here (PDF).

Interested in additional clinical trials? For more information about PKD clinical trials, click here.