title: A potential new prodrug for the treatment of cystinosis: design, synthesis and in-vitro evaluation.
abstract: Nephropathic cystinosis is a rare autosomal recessive disease characterised by raised lysosomal levels of cystine in the cells of most organs. The disorder is treated by regular administration of the aminothiol, cysteamine, an odiferous and unpleasant tasting compound that along with its metabolites is excreted in breath and sweat, leading to poor patient compliance. In an attempt to improve patient compliance a series of novel prodrugs has been designed and evaluated as a potential new treatment for nephropathic cystinosis. The first of the prodrugs tested, 3a, was found to decrease the levels of intracellular cystine in cystinotic fibroblasts. This is the first report of a potential new therapeutic treatment for nephropathic cystinosis since the advent of cysteamine bitartrate.
authors.affiliation: School of Pharmacy, The Robert Gordon University, Scotland, Aberdeen, Aberdeenshire AB10 1FR, UK.
authors.forename: Bridgeen
authors.initials: B
authors.lastname: McCaughan
authors.forename: Graeme
authors.initials: G
authors.lastname: Kay
authors.forename: Rachel M
authors.initials: RM
authors.lastname: Knott
authors.forename: Donald
authors.initials: D
authors.lastname: Cairns
mesh_terms: D002478:Cells, Cultured; D003543:Cysteamine / Q000031:analogs & derivatives* / Q000737:chemistry / Q000494:pharmacology*; D003554:Cystinosis / Q000188:drug therapy*; D004847:Epithelial Cells / Q000187:drug effects; D005347:Fibroblasts / Q000187:drug effects; D006801:Humans; D015394:Molecular Structure; D011355:Prodrugs
chemical_list: D011355:Prodrugs; D003543:Cysteamine