Design, Synthesis and Initial In Vitro Evaluation of Novel Prodrugs for the Treatment of Cystinosis
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
Pages 336-345 (10)
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.
Cystinosis, prodrugs, GGT, cysteamine, cystamine
Sunderland Pharmacy School, University of Sunderland, Sunderland, SR1 3SD, UK.