A drug target for focal segmental glomerulosclerosis

Focal segmental glomerulosclerosis (FSGS) is a syndrome for which there is no reliable treatment, in part because of a poor understanding of its pathophysiology. However, recent research has identified a potential target for therapy.

Figure. This PAS-stained section shows the perihilar location of sclerosis with hyalinosis and lipids vacuolation and an adhesion to Bowman's capsule, which are characteristic features of FSGS. Reproduced with the permission of Dr. J. Charles Jennette.

FSGS is characterized clinically by varying degrees of proteinuria; 50% of patients progress to end-stage renal disease within 10 years. On the basis of research involving a large family with hereditary FSGS, Winn and colleagues have identified a shared genetic mutation that results in an increase in the amount of calcium transported into glomerular cells.1 Since ion channels are easily inhibited through drug therapy, the authors of the study suggest that the modified protein may be a good target for the treatment of chronic kidney disease.