Polycystic kidney disease (PKD) is a genetic disorder portrayed by the development of several cysts in the kidneys. A research from Yale University claims that an anti-convulsant drug usually applied to treat epilepsy could decrease cysts in mice that are linked to PKD.
Approximately more than 6, 00,000 people in the US apparently suffer from this kidney disease.
“This is exciting because the drug (valproic acid) is also in clinical trials as a potential cancer drug and has a known safety profile,” commented, Zhaoxia Sun, associate professor of genetics and senior author of the paper.
Sun’s lab apparently started looking for chemical compounds that could curb the consequences of one of two genes identified to cause the most frequent type of PKD, which is alleged to be the fourth top cause of kidney failure. The screens supposedly recognized a group of molecules known as HDAC inhibitors as likely drug applicants. These inhibitors are said to remove the curve tail in zebrafish, a shortcoming apparently linked to the PKD-causing gene.
The epilepsy drug valproic acid, promoted under brand names like Depakote, is also believed to be a HDAC inhibitor. So Sun’s colleague Stefan Somlo, professor of internal medicine and genetics at Yale School of Medicine, supposedly utilized the drug to treat mice suffering from PKD. The team observed that treated mice apparently exhibited a noticeable decrease of cysts and enhanced kidney function.
Sun remarked, “Currently there is no directed therapy, but it is a slow progressing disease, which frequently takes decades to develop symptoms. Therefore, a treatment of even modest efficacy could have significant clinical impact.”
Sun noted that numerous HDAC inhibitors are apparently being crafted as anti-cancer drugs and each could be a possible applicant to treat PKD.
The research was published in the Proceedings of the National Academy of Sciences.