On June 9th Chairman, Roy Forsyth, his wife Vicky and Secretary, John Terry, visited the School of Pharmacy and Life Sciences at the Robert Gordon University (RGU). Our host was Professor Don Cairns who moved to RGU from Sunderland about 7 years ago. During that time Don has built up a strong group of scientists working on various ways to tackle the problem of delivering cysteamine (the active in Cystagon) to patients in more effective/less unpleasant ways. We met all of the team during our visit and were taken on a tour of the impressive facilities by Dr Graeme Kay.
Don brought with him from Sunderland a strong interest in Prodrugs. Prodrugs are compounds that contain an active drug combined with other chemicals in a way that allows it to pass into the body to where it is needed without being degraded in the stomach or liver, and alleviating any bad effects on stomach linings, for example. We have been supporting work on Prodrugs at Sunderland since Don’s time there but he has continued parallel work, using different chemical approaches, at RGU. We have provided support: purchasing necessary equipment for this work.
As at Sunderland a lot of effort has to go into the proof that these new chemicals do work in the way intended. This is also true at RGU where the biological studies are led by Dr Rachel Knott. Some months back we were asked to fund this side of the work but at the time did not have sufficient funds to help. This underlines the importance of fundraising: there are always more worthwhile projects to fund than we can support. Because we were unable to help there are a set of Prodrugs waiting bio-evaluation at RGU.
In parallel to the Prodrug work the team is also looking at alternative means of delivering Cysteamine without modification. This part of the effort is carried out by Barbara Buchan and we have funded all her work to date. She has produced suppository formulations which have shown excellent dissolution characteristics in the lab and have good stability. She has also worked on eye gels where the aims are sustained release of cysteamine over a much longer time than is experienced with the eye drops and reduced loss through drainage from the eye partly through some built-in bioadhesion. Again, laboratory tests are positive. Finally, she is looking at the possibility of delivering the drug by a puffer as used by asthmatics. By producing tiny microspheres of cysteamine with a biodegradable coating this may be possible. The lungs have a huge surface area and, if the cysteamine will go through the lung walls, it would be delivered directly into the blood stream. We are funding a visit by Barbara to a conference in the USA where she has been invited to present her work.
This was an interesting, stimulating visit and we intend to try to make it a regular event.
John Terry, Cystinosis Foundation UK