SCIENTISTS on Wearside have won backing from America for their work to combat a rare genetic condition.
Pharmaceutical research being carried out at Sunderland University’s new multi-million pound science complex could hold the key to curing cystinosis.
The Echo told yesterday how 20-year-old Sarah Laing, who has the condition, is praying for a new kidney after her body rejected a donor organ, and she is now on dialysis four times a week.
Now, as a result of ongoing research, the Wearside pharmaceutical scientists have won £80,000 funding from America to carry out a comprehensive three-year study into the disease using the latest research techniques.
The successful bid for funding from the U.S. charity Cystinosis Research Network (CRN) will see Professor Roz Anderson head up a team of academics to learn more about the disease.
They aim to identify biomarkers to help improve detection and early diagnosis, as well as develop new targets for intervention and preventative treatment, offering new hope to thousands of sufferers and their families.
Sarah, of Moorside, Sunderland, who was diagnosed with cystinosis when she was 18-months-old, said: “This is fantastic news. I don’t know if it will be able to benefit me now, but it would be great if this research could help others in the future.”
Prof Anderson said: “This is an incredibly exciting project to be involved in.
“Although there has been some very good research carried out on this disease in the past, there’s not been a systematic study until now and we are at an advantage of using the latest techniques, so we’re hopeful of discovering new pathways to intervention.”
The study will focus on the condition of the inherited disease at a cellular level using the latest “proteomic” techniques - the study of proteins in cells.
Currently, cystinosis is treated with the active ingredient cysteamine (marketed as Cystagon®), but, this produces unpleasant side effects such as nausea, vomiting, severe halitosis, body odour and a noxious taste.
A large dose of the drug is required four times a day to treat the disease, as up to 70 per cent is wasted through the body’s metabolism during digestion and absorption.
However, the university team has already modified the drug to target cysteamine directly into the cells, like a ‘magic bullet’, aiming to improve its absorption into the body, reduce the amount needed to be taken and lower the adverse side effects.
This research continues alongside the new project and is funded by the British charity, Cystinosis Foundation UK.
But, the CRN funding will help PhD researcher Jill Jobson, a Sunderland graduate of chemical and pharmaceutical Science, investigate further the effects cysteamine has on the cystinotic cells and how it’s treating the disease.
Christy Greeley, CRN president and executive director, said: “We are proud to support the groundbreaking work of Prof Anderson and her associates at Sunderland.”