Dr Don Cairns, Robert Gordon University
I have been working on cystinosis for about five or six years, initially at the University of Sunderland and now at The Robert Gordon University (RGU) in Aberdeen, where I am the Associate Head of the School of Pharmacy.
The work at RGU is designed to improve the quality of life of cystinosis sufferers by making prodrugs of cysteamine. Prodrugs are compounds which are inactive when taken, but once within the body the prodrug is converted into an active form which acts to treat the condition.
The prodrugs we are designing at RGU will be odourless, tasteless and active when taken by mouth. Ideally, we want the compounds to remain in the body for several hours to allow patients to take the medication only once or twice daily rather than the six times a day dosing used at the moment.
We are trying to eliminate the unpleasant taste and smell of cysteamine by making water-insoluble, non-volatile derivatives of the drug. A drug can only taste bad if it dissolves in the saliva in your mouth; similarly, if the drug is not volatile it cannot smell bad. The techniques adopted at RGU are not new and they have been widely used in the pharmaceutical industry to disguise the bitter taste of antibiotics such as erythromycin and chloramphenicol. This means that the toxicity data for these compounds have already been established in many cases, so we hope the lead time required to produce a new treatment will be reduced. We believe that this approach yields the best chance of developing a new treatment for cystinosis in the shortest possible time.
The research at RGU is funded by Cystinosis Foundation Ireland and a number of charities local to the North East of Scotland.