UC Santa Barbara researchers, in collaboration with the biotech firm, Endocyte, have put a new twist on a common drug, which could successfully treat PKD in the future. In earlier research, UCSB scientist Thomas Weimbs had identified the immunosuppressant drug rapamycin as a good candidate for PKD treatment. While rapamycin effectively stopped PKD progression in mice, clinical trials showed that safe levels of the drug weren’t enough to stop kidney cysts in humans.
In this latest research, the scientists decided to find out if they could target PKD cysts in the same way they target cancer cells. Endocyte has successfully added folate to cancer drugs, thus delivering the treatment straight to cancer cells with folate receptors. The team then found that both mouse and human PKD cysts do, indeed, have folate receptors. Endocyte created the new drug, FC-rapa, which links folate with rapamycin and directly targets the kidneys. Treatment in mice was promising, preventing PKD cyst growth with fewer side effects.
New Drug Shows Promise for Kidney Disease, ScienceDaily, August 2, 2012