title: Synthesis and in vitro evaluation of novel pro-drugs for the treatment of nephropathic cystinosis.
abstract: As part of our continuing work to obtain new pro-drugs for the treatment of nephropathic cystinosis, a number of glutaric and succinic acid derivatives of cystamine have been designed, synthesised and biologically evaluated in vitro. These compounds have been designed as odourless and tasteless pro-drugs which will release multiple molecules of cysteamine upon administration. All of the synthesised compounds evaluated in this study were non-cytotoxic and displayed a greater ability than cysteamine to deplete the levels of cystine in cultured fibroblasts.
authors.affiliation: School of Pharmacy and Life Sciences, Robert Gordon University, Schoolhill, Aberdeen AB10 1FR, UK. z.omran@rgu.ac.uk
authors.forename: Ziad
authors.initials: Z
authors.lastname: Omran
authors.forename: Kevin A
authors.initials: KA
authors.lastname: Moloney
authors.forename: Amina
authors.initials: A
authors.lastname: Benylles
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: D003538:Cystamine / Q000031:analogs & derivatives* / Q000627:therapeutic use / Q000633:toxicity; D003554:Cystinosis; D005198:Fanconi Syndrome; D005347:Fibroblasts / Q000187:drug effects; D005977:Glutarates / Q000737:chemistry; D006801:Humans; D009404:Nephrotic Syndrome / Q000188:drug therapy*; D011355:Prodrugs / Q000138:chemical synthesis* / Q000627:therapeutic use / Q000633:toxicity; D019802:Succinic Acid / Q000737:chemistry
chemical_list: D005977:Glutarates; D011355:Prodrugs; D019802:Succinic Acid; C035736:glutaric acid; D003538:Cystamine