Cysteamine prodrugs for an improved treatment of cystinosis

WHAT IS A PRODRUG? Prodrugs can be designed to overcome a range of problems, including inefficient delivery, poor absorption, poor bioavailability and noxious taste and smell . They are inactive forms of a successful clinical drug which are activated, usually by an enzyme, once inside the body to release the active parent drug, Figure 1. Our initial work with simple prodrugs demonstrated proof of concept [1] and we are now carrying out full evaluation of a series ofpossible clinical candidates.