How worms' sex behavior can have a major impact on understanding human disease.

T he “Golden Fleece Award” was established by the late Senator William Proxmire in 1975 to call attention to wasteful federal spending on useless and seemingly inane research. No doubt, enormous waste and corruption routinely emanate from Washington, DC. However, this and other eff orts to expose such evil sometimes have the eff ect of trivializing basic scientifi c inquiry that has no obvious and immediate benefi t to man. For example, a Golden Fleece was awarded to a study of the sex life of the screw-worm fl y. After the senator’s criticism of this $250,000 research project, the results were used to create sterile screw-worms that were released into the wild and eliminated this major cattle parasite from the US. Th is eff ort saved the US cattle industry about $20 billion. Although it did not win a Golden Fleece Award, the more recent study of the mating behavior of the roundworm Caenorhabditis elegans seemed like another waste of time and money to some underinformed individuals. However, no one could have predicted that this X-rated worm research could have a profound impact on our understanding of an important and severe human disorder—autosomal dominant polycystic kidney disease (ADPKD)—and lead to new treatments for patients with this disorder. ADPKD is the most common form of the genetic cystic kidney diseases; it aff ects more than 12 million patients worldwide and 600,000 in the USA. Th e disorder is characterized by the development of innumerable cysts in the kidneys and other organs, including the liver, and eventual dysfunction of the aff ected organs. Most patients with ADPKD will eventually develop kidney dysfunction and require dialysis or transplantation. Some succumb to ruptured cerebral aneurysms, which develop in about 10% of the patients. As genetic research progressed, the gene found to be mutated in this disease was identifi ed and called pkd-1. (Th is gene accounts for about 80% of the adult form of this disease. Th e other 20% is caused by a diff erent gene, pkd-2.) Th e protein product of the gene was identifi ed and named polycystin. However, for some time, no one knew what this protein did or how its mutated state produced kidney problems. Th en some answers came from research into the sex life of the round worm C. elegans (Figure). Th is particular roundworm is a very popular and famous worm among many worm researchers (yes, there are many). C. elegans has certain charFrom the Division of Nephrology, Department of Internal Medicine, Baylor University Medical Center at Dallas.