Pyrimethamine, an antiparasitic compound, has been further tested in ADPKD preclinical models and was found to decrease cell proliferation in ADPKD patient-derived cultured epithelial cells and prevent renal cyst formation in Pkd1-KO mice (Takakura et al. 2011). The gene discussed is PKD1; the disease is autosomal dominant polycystic kidney disease.