PKD: cease and de-cyst
Marketed treatments for polycystic kidney disease manage symptoms but do not slow disease progression. A research team led by Genzyme Corp. now has shown that blocking biosynthesis of the glycosphingolipid glucosylceramide could help treat the underlying causes of PKD.1 The findings could represent a new indication for Genzyme's eliglustat tartrate, a glucosylceramide synthase inhibitor that is in Phase III testing for Gaucher's disease.
The most common causes of PKD are mutations in polycystic kidney disease 1 (PKD1) or PKD2, both of which code for proteins that are expressed in the cilia of renal epithelial cells. Although different PKD mutations can produce a variety of symptoms, all forms of the disease are characterized by the development of cysts in the kidneys that compromise the organ's