Tyrosine Kinase Inhibitor Slows Cyst Growth in Autosomal Dominant Polycystic Kidney Disease (ADPKD) in New Study

A cancer drug called bosutinib may inhibit the growth of cysts in patients with autosomal dominant polycystic kidney disease (ADPKD), according to a study published online on August 24, 2017 in the Journal of the American Society of Nephrology (JASN). The JASN article is titled “Bosutinib Versus Placebo for Autosomal Dominant Polycystic Kidney Disease." The findings point to a potential new treatment strategy for affected patients, but the long-term benefits remain to be determined. ADPKD is an inherited disorder that affects up to 1 in 1000 people and is characterized by cysts in the kidney and other organs. As patients' kidney volume increases due to cyst growth, they gradually lose their kidney function and often develop kidney failure. Current treatments are primarily supportive, such as focusing on hypertension and other secondary complications. The inherited mutations that cause ADPKD affect a protein involved in various signaling pathways that often involve enzymes called tyrosine kinases. Therefore, a team led by Vladimir Tesar, MD, PhD (Charles University and General University Hospital, in the Czech Republic) tested the potential of an investigational drug called bosutinib that inhibits a particular tyrosine kinase called Src/Bcr-Abl.
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