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UK Researchers Granted $131K for Proteomics-directed Cystinosis Research

By a GenomeWeb staff reporter

NEW YORK (GenomeWeb News) – The University of Sunderland, UK has received a three-year £80,000 ($131,424) grant from the Cystinosis Research Network to perform proteomics research into the disease in order to find better treatments.

The research will be led by Roz Anderson, a professor in the university's department of pharmacy, health, and well-being, and will investigate cystinosis at the cellular level, using the latest proteomic technologies, the school said. Work will be conducted at the university's new £7.5 million science complex, which opened in February.

Collaborating on the research will be Achim Treumann at the North East Proteome Analysis Facility in Newcastle, UK.

Cystinosis occurs when the body fails to remove excess cystine. There is no cure for the disease and if left untreated, it can lead to kidney failure as well as other organ problems before a patient reaches the age of 10. Until recently, patients rarely lived beyond the age of 20, the university said.

The disease is currently treated with cysteamine, which has side effects that include nausea, vomiting, extreme bad breath, body odor, and a noxious taste. Though large doses of the medication are required to be taken by patients four times daily, as much as 70 percent of it provides no therapeutic benefits, because the body fails to digest and absorb the drug completely.

Anderson and her colleagues have modified the drug, however, to target cysteamine directly into the cells, which would increase absorption while reducing side effects and the dosage required. The funding from the Cystinosis Research Network will be used to further research the effects of cysteamine.

"Although there has been some very good research carried out in 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," Anderson said in a statement.

In addition to treating cystinosis, cysteamine has also been shown to have therapeutic potential as a treatment for Huntington's disease and Alzheimer's disease, and the research may result in the development of the drug for treatment in those indications, Anderson added.

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