SCIENTISTS have been handed £125,000 to carry out breakthrough research into a rare genetic disease.
The medical team from Sunderland University are trying to improve the lives of millions of cystinosis sufferers.
There is no cure for the condition, which is triggered due to a patient’s inability to produce the amino acid cystine.
This can cause malfunctioning in cells, leading to kidney problems.
The funding, from the Cystinosis Foundation UK (CFUK), will support research evaluating whether prodrugs, intended to drastically improve the condition, could work effectively.
Cystinosis is treated with the active ingredient Cysteamine, but this produces unpleasant side effects, such as nausea, vomiting, halitosis, body odour and a noxious taste.
A large dose of the drug is required four times a day, as up to 70 per cent is wasted through the body’s metabolism.
But with ongoing support from CFUK, the university team has been able to design prodrugs to take the effective treatment directly to the cells that need it, improve its absorption and reduce the unpleasant side effects.
Professor Roz Anderson, who heads up the team of pharmaceutical scientists, said: “Initial tests have shown that these prodrugs have the potential not only to produce the required therapeutic effects, but also to reduce the side effects and improve the patient experience.
“This new financial support will allow us to work with clinicians and industrial collaborators in Europe to investigate the prodrugs for their potential to progress to patient use.”
Prof Anderson is supported in her team by Dr Lisa Frost, Paul Hambleton and Dr Hamde Nazar.
It is hoped that the research will lead to an effective and improved treatment for the condition.
In addition to the drug research, PhD graduate Dr Frost has been testing drugs designed to treat eye problems associated with cystinosis.
One of the consequences of the disease is a build-up of cystine crystals in a sufferer’s eyes, causing pain, discomfort and photophobia.
The new drugs are designed to improve the current treatment.