On October 2nd, Vicky and Roy Forsyth, of Mintlaw Aberdeenshire, met up with Professor Don Cairns and his team at the Robert Gordon University in Aberdeen to see first hand the research they have been performing in order to improve the medicines Cystinosis sufferers must take. Vicky and Roy’s 5-year-old daughter, Lena, has Cystinosis and Roy is a member of the Cystinosis Foundation UK committee.
Professor Donald Cairns BSc is the Associate Head of School and MPharm Programme Director in the School of Pharmacy at The Robert Gordon University. His team are currently funded by the Cystinosis Foundation Ireland and have also received grants for new equipment, such as an oven (pictured), from the UK Foundation. Professor Cairns tells us about the research he and his team are undertaking:
"The cystinosis research at RGU is designed to produce an orally active treatment for cystinosis that doesn’t taste bad, doesn’t smell bad and can be given once or twice daily. We are trying to achieve this by making a number of prodrugs of cysteamine. Prodrugs are pharmacologically inert i.e. they themselves do not work in the body, but once they have been absorbed, the prodrug decomposes to release the active cysteamine.
The prodrug approach has been used successfully in a number of therapeutic areas including antibiotics, antihistamines and ulcer treatments.
The advantage of using prodrugs is that the cysteamine is chemically ‘camouflaged’ and no cysteamine is released until the drug has passed out of the gut and into the cells of the body. We hope that in this way the stomach upsets, bad breath etc. associated with cysteamine will be minimised or even abolished entirely.
So far about twenty odourless and tasteless prodrugs have been made. These compounds are currently being tested to see if they lower the high levels of cystine found in cystinotic cells. The work is still in its early stages, but initial results have been very encouraging.
The prodrug work at RGU is funded by Cystinosis Foundation Ireland and we are grateful for their support."
Don Cairns and his team (left to right): Graeme Kay, Don Cairns and Bridgeen McCaughan
The oven provided by Cystinosis Foundation UK
Both Vicky and Roy thank Professor Cairns and his team for their time and we all wish them the very best with their research endeavours.