Research Today

Abstract

Basic research has identified the 2 major defects linked to polycystic disease: (i) the cystic cells proliferate excessively; (ii) these cells secrete fluid that enlarges the cysts. The main therapeutic strategies in autosomal dominant polycystic kidney disease (ADPKD) are the use of drugs capable of interfering with the mechanisms causing these defects. One of the strategies explored was the inhibition of the mTOR system. Unfortunately, 2 clinical trials have failed to show a protective effect of this class of drugs. Somatostatin is another molecule under intense clinical validation. At the moment, the collected data suggest a possible activity against ADPKD, but the results are still too preliminary to attain clinically meaningful conclusions. Tolvaptan is a vasopressin receptor antagonist that has been extensively studied: a clinical trial recruiting an adequate population suggested a possible positive effect of this molecule in the reduction of renal volume growth and the achievement of significant clinical targets. The near future promises new exploratory clinical trials of molecules that have already been assessed in the recent past or of completely new therapeutic strategies. For the high number of enrolled patients, the HALT study attracts the attention of the scientific community. This trial will explore the role of antagonists of the renin-angiontensin system in slowing the progression of ADPKD. Finally, a category of drugs previously unexplored is that of the inhibitors of the epidermal growth factor receptor. Clinical research in ADPKD is extraordinarily active in this period and this consideration allows a cautious optimism about the prospective therapies for this disease that for long remained orphan. A shadow on the prospect of future results in clinical research in this field comes from the observation that a considerable number of trials show a methodologically inadequate design.