Modified Rapamycin Shows Promising Results for Polycystic Kidney Disease Model

Scientists at University of California - Santa Barbara have demonstrated in the laboratory that modification of the drug rapamycin, often used as a post-transplant anti-rejection drug, is effective in treating polycystic kidney disease (PKD) in cystic animal models. The findings resulted from collaboration between UCSB and Endocyte, Inc., a biotech firm based in Indiana. The study was published in last week's Journal of the American Society of Nephrology.The study was published in last week's Journal of the American Society of Nephrology.

The research effort was directed by Thomas Weimbs, Associate Professor in the Department of Molecular, Cellular, and Developmental Biology at UCSB. He has been studying the disease for more than a decade. The current findings build on research performed in 2006 in the Weimbs lab, showing that the drug, which has been in use for years as an immunosuppressive agent, was highly effective in stopping disease progression in mouse models of polycystic kidney disease.

Because of its ability to slow disease progression in animal models of PKD, rapamycin was evaluated in several human clinical trials in recent years with mixed results. The dosage of the drug in a large Novartis study proved to be too low to affect cyst growth. Endocyte, Inc., a small biotech firm specializing in developing cancer drugs, collaborated with Dr. Weimbs to chemically synthesize a modified version of rapamycin by conjugating it with folate (FC-rapa). ‘FC-rapa is a fascinating drug because it combines an extremely specific drug with a delivery approach that targets it to a specific organ, the kidney,’ said Weimbs. When this new drug was tested in cystic mice, it was found to still be highly effective in preventing kidney cyst growth and had fewer side effects. It will be a few years before it may progress to clinical testing in humans.

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