Health & Medicine - Posted by Bill Hathaway-Yale on Tuesday, December 8, 2009 11:13 - 2 Comments 
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(No Ratings Yet) Loading ...Seizure med may combat kidney condition
“Currently there is no directed therapy, but it is a slow progressing disease, which frequently takes decades to develop symptoms,” lead researcher Zhaoxia Sun says. “Therefore, a treatment of even modest efficacy could have significant clinical impact.”
YALE (US)—An anticonvulsant drug commonly used to treat epilepsy reduces cysts in mice that are associated with polycystic kidney disease (PKD), a difficult-to-treat ailment that afflicts 600,000 people in the United States.
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The text of this article by Futurity is licensed under a Creative Commons Attribution-No Derivatives License.
“This is exciting because the drug (valproic acid) is also in clinical trials as a potential cancer drug and has a known safety profile,” says lead researcher Zhaoxia Sun, associate professor of genetics at Yale University.
Sun’s lab began searching for chemical compounds that would suppress the effects of one of two genes known to cause the most common form of PKD, which is the fourth leading cause of kidney failure. The screens identified a class of molecules called HDAC inhibitors as potential drug candidates. These inhibitors eliminated the curve tail in zebrafish, a defect associated with the PKD-causing gene.
The epilepsy drug valproic acid, marketed under brand names such as Depakote, is also a HDAC inhibitor. So Sun’s collaborator Stefan Somlo, professor of internal medicine and genetics at Yale School of Medicine, used the drug to treat mice with PKD. The team noted that treated mice showed a marked reduction of cysts and improved kidney function.
Sun observed that multiple HDAC inhibitors are being developed as anti-cancer drugs and each would be a potential candidate to treat PKD.
“Currently there is no directed therapy, but it is a slow progressing disease, which frequently takes decades to develop symptoms,” Sun says. “Therefore, a treatment of even modest efficacy could have significant clinical impact.”
The paper published online in the Proceedings of the National Academy of Sciences. Edward Mallinckrodt Jr. Foundation and the National Institute of Diabetes and Digestive and Kidney Diseases funded the study.
Yale University news: http://opa.yale.edu/
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2 Comments
Randall S. Fryar
QUIANA CARRINGTON
IT WOULD BE GREAT IF THIS CAN BE A POSSIBLE TREATMENT FOR THIS CONDITION. I WAS DIAGNOSED WITH OKD AT THE AGE OF 10 AND HAVE HAD PROBLEMS EVER SINCE. IN THE RIGHT PERSON, THIS DISEASE CAN BE PAINFUL AT A VERY YOUNG AGE AND I WOULD DIE INSIDE IF MY CHILDREN ARE ONE DAY DIAGNOSED WITH IT. PLEASE HELP PEOPLE WITH PKD TRY TO GET THROUGH THIS AND LIVE LONG LIVES
The potential of volproic acid in combatting PKD sounds exciting. Given the state of research, how long might it take to find itself in a prescription form for the thousands suffering from this disease (best guess)?