Title: Design, Synthesis and Initial In Vitro Evaluation of Novel Prodrugs for the Treatment of Cystinosis
VOLUME: 3 ISSUE: 5
Author(s):R. J. Anderson, D. Cairns, W. A. Cardwell, M. Case, P. W. Groundwater, A. G. Hall, L. Hogarth, A. L. Jones, O. Meth-Cohn and P. Suryadevara
Affiliation:Sunderland Pharmacy School, University of Sunderland, Sunderland, SR1 3SD, UK.
Keywords:Cystinosis, prodrugs, GGT, cysteamine, cystamine
Abstract: Simple amino acid prodrugs of cysteamine and cystamine were synthesized to overcome the problems of palatability and gastrointestinal irritation, while γ-glutamyl prodrugs were synthesized to target the parent drug to γ-glutamyl transpeptidase (GGT). Preliminary cystine-depleting evaluation in vitro suggests further investigation is warranted. Low toxicity of the prodrugs was observed.