Chloride channel opener

Chloride channel openers refer to a specific category of drugs designed to modulate chloride channels in the human body. Chloride channels are anion-selective channels which are involved in a wide variety of physiological functions and processes such as the regulation of neuroexcitation, transepithelial salt transport, and smooth muscle contraction.^[1] Due to their distribution throughout the body, diversity, functionality, and associated pathology, chloride channels^[2] represent an ideal target for the development of channel modulating drugs such as chloride channel openers.

Chloride channel modulators include chloride channel openers, and chloride channel blockers, both of which modulate the transport of chloride ions through chloride channels.^[3] Chloride channel openers specifically work by either preventing the closure of chloride channels, or promoting their opening, thus helping to facilitate movement of chloride ions into a cell.

In general, mutations in various chloride channels throughout the human body can result in many pathologies, such as macular degeneration, myotonia, cystic fibrosis, and hyperekplexia.^[3] Chloride channel openers have been proposed to treat a variety of such pathologies, most notably including cystic fibrosis, a genetic disorder in which a defect in the cystic fibrosis transmembrane conductance regulator protein impacts chloride ion transport across epithelial cells.^[4]

Chloride channels remain a somewhat under-explored target for drug development despite their biological significance, due to various issues associated with the development of successful chloride channel drugs. As a result, the development of chloride channel modulators such as chloride channel opening drugs has been limited. The relative structural complexity of chloride channels, their varied roles in biological processes, and the problems associated with the development of drugs with high specificity emphasize the necessity for further research in this area.