Charitable funding for cystinosis research
31 Jan 2011 by Evoluted New Media
A chemically modified version of drug used to treat cystinosis could spell the end of its unpleasant side effects say researchers from the University of Sunderland who have received funds for new equipment and a PhD student.
A chemically modified version of drug used to treat cystinosis could spell the end of its unpleasant side effects say researchers from the University of Sunderland who have received funds for new equipment and a PhD student.
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Professor Roz Anderson and PhD student Lisa Frost received funding and equipment from Cystinosis charity to research the disease. |
Professor Roz Anderson and PhD student Lisa Frost – whose PhD is being funded by Cystinosis Foundation UK – have modified a drug used to treat cystinosis, a rare genetic disease in which the body’s mechanism to remove excess cystine breaks down, leading to kidney problems.
A large dose of the drug is currently required four times a day as 70% is wasted through the body’s metabolism during digestion with no therapeutic effects. This can cause nausea, vomiting, severe halitosis, body odour and a noxious taste.
Anderson and Frost have chemically modified a drug containing the active ingredient cysteamine to target gamma-glutamyl transpeptidase, an enzyme on the surface of most cells that will carry the modified drug into the cell.
“It becomes more like a magic bullet,” Anderson said, “Getting directly inside those cells where it needs to be before the drug is released, increasing the therapeutic benefit. This means the patient needs less of the drug, as the dose is not being wasted through metabolism.”
Anderson said the initial work shows the pro-drug approach can produce the required medicinal action and is currently assembling an international team of experts to investigate the mechanism of how the drug works.
As well as funding Frost’s PhD, Cystinosis Foundation UK raised £14,000 to buy the university High-Performance Liquid Chromatography (HPLC) equipment.
“We are committed to funding Lisa’s work and research on the drug,” said Roy Forsyth, chairman of the foundation, “We realised she needed the HPLC equipment to make her work more effective and efficient. It’s crucial the research happens quickly and effectively as possible; it will change lives.”
“The HPLC equipment will certainly help with the project; it’s new, reliable and dedicated to my work,” said Frost, “Meeting the families and children affected by this disease really pushed home how vital the work we are doing here is and made me even more determined to get results.”
