Moreover, the evidence that mTOR deregulation participates in NF1‐related tumorigenesis and the recent finding that mTOR and its downstream effector S6K play a critical role in the pathogenesis of ADPKD, support the possibility that rapamycin could represent a viable therapy for patients with this rare association (Hegedus et al., 2008; Johannessen et al., 2005, 2008; Shillingford et al., 2006; Wahl et al., 2006; Wen & Kesari, 2008). The gene discussed is MTOR; the disease is autosomal dominant polycystic kidney disease.