A group of renal protective compounds active in PKD that had been identified include paclitaxel, taxanes, brefeldin-A, TNF-a and TNF-a inducing agents. In vivo data using cpk mice, a murine model for PKD, showed extended survival for animals treated with taxol as compared with controls. The gene mutated in cpk mice has been found to code for a novel protein called cystin, which is located in the mono cilium on epithelial cells. Polycystin-1 and polycystin-2, the gene products of PKD1 and PKD2, the two genes most commonly mutated in human PKDare also found in the mono-cilium suggesting possible interaction between cystin and polycystin-1 and -2. The tubulin subunit of microtubules is the specific target of taxol and active taxanes. Microtubules are now known to be intimately involved in the functions of several PKD gene products. However, how taxol and related taxanes protect against PKD remains unclear.

In addition, researchers at UCLA have developed an in vitro model for preclinical screening of compounds effective in treating PKD. The screening system, comprising PKD cells that form cysts in vitro, is currently the only cell culture system available for identifying drug candidates.

Reference: UCLA Case Nos. 1996-525; 1992-505

US Patent Numbers: 6,011,055 5,882,881 5,789,189 5,750,495

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