An experimental drug called roscovitine may inhibit a degenerative kidney disease that so far has defied cure, a study in mice shows.
Combined with promising results from animal studies on other potential drugs, the new finding brightens the outlook for people with the inherited condition called polycystic kidney disease (PKD). The disease, whose symptoms often don't arise until adulthood, usually causes back pain, high blood pressure, urinary tract infections, and, ultimately, kidney failure. Other than a complete-kidney transplant, available treatments address only the disease's symptoms.
People with PKD have a mutation in one of two genes that encode proteins called polycystins. These proteins are necessary for the proper functioning of organelles called cilia that protrude from kidney cells. One hypothesis holds that cilia sense the flow and composition of fluids in the kidneys. The cilia rely on polycystins to relay this information into a cell and regulate its dev