New Drug Trial for PKD

It has been found out that the drug rapamycin is effective in controlling the progress of polycystic kidney disease (PKD) in mice. Rapamycin has long been used as an immunosuppressant, however recent study has found that it can inhibit the growth of the cysts.

We all know that PKD is a hereditary disease which will accompany the patients for lifelong time. In the United States there are more than 600,000 people suffering from PKD and it is one of the leading causes of renal failure.

The finding came from the collaboration of Weimbs and Leamon. In an earlier study of Weimbs, he found that a signaling protein called mTOR can stimulate the cyst growth in PKD. And then Weimbs found that rapamycin can affect this protein and prevent the growth of cysts in PKD.

The drug trial on mice achieved very good effect, but unfortunately the dose at which it can be safely applied on human proved to be too low to affect the cysts.

Through the combination with folate, a new version of rapamycin called folate-conjugated rapamycin was created. It can still stop cyst growth and at the same cause less side effects.

This drug trial has been proved very effective on mice, but it seems not very satisfying on human. Since Leamon himself has PKD and he begun to take regular rapamycin with his doctor’s permission. The drug was ineffective and he eventually developed into renal failure. Fortunately he received a kidney from his sister and he is now in good health.

Even though, Leamon is still committed to this study and they will continue the tests of folate-conjugated rapamycin and he hopes to find an effective drug for treating PKD. We are all expectant for that day!