Since the discovery of aquaporin-1 (AQP1) as a
water channel, more than 2,000 articles, reviews and chapters have been published. The wide tissue expression, functional and
biological roles have documented the major and essential physiological importance of these channels both in health and disease. Thus, over the years, studies have revealed essential importance of
aquaporins in mammalian pathophysiology revealing
aquaporins as potential
drug targets. Areas covered: Starting from a brief description of the main structural and functional features of
aquaporins, their roles in physiology and pathophysiology of different human diseases, this review describes the main classes of small molecules and
biologicals patented, published from 2010 to 2015, able to regulate AQPs for diagnostic and therapeutic applications. Expert opinion: Several patents report on AQP modulators, mostly inhibitors, and related
pharmaceutical formulations, to be used for treatments of water imbalance disorders, such as
edema. Noteworthy, a unique class of
gold-based compounds as selective inhibitors of
aquaglyceroporin isoforms may provide new chemical tools for therapeutic applications, especially in
cancer. AQP4-targeted
therapies for
neuromyelitis optica, enhancement of AQP2 function for
nephrogenic diabetes insipidus and AQP1-5 gene transfer for the
Sjogren's syndrome represent promising
therapies that deserve further investigation by clinical trials.