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Bending the curve: drug halts kidney cysts

Medicine@Yale, 2010 - Jan Feb


About one-half of patients with the most common form of polycystic kidney disease (PKD), which affects about 600,000 Americans, will eventually succumb to kidney failure requiring transplant or dialysis.).

Researchers studying zebrafish have noted that kidney cysts in these animals are often accompanied by a distinctive body curvature (see photo). Using this curve as a guide, Associate Professor of Genetics Zhaoxia Sun, Ph.D., and colleagues report in the December 22, 2009 issue of Proceedings of the National Academy of Sciences that kidney cysts could be suppressed in the fish with valproic acid, a drug used for epilepsy and bipolar disorder under the trade name Depakote. The effectiveness of the compound was then confirmed in mice by PKD expert Stefan Somlo, M.D., the C.N.H. Long Professor of Internal Medicine.

“This is exciting,” says Sun, “because valproic acid is also in clinical trials as a potential cancer drug and has a known safety profile.”

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