Revolutionary treatment could help Lena

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A MINTLAW schoolgirl could benefit from a new treatment for a rare disease, after researchers at Aberdeen’s Robert Gordon University received a cash boost.

Lena Forsyth (10), was born with a rare genetic metabolic condition called Cystinosis, which can lead to liver problems and kidney failure. The condition is caused by a build-up of an amino acid called cystine which is usually broken down by the body.

But in cystinosis sufferers, it accumulates in all the body’s organs, including the eyes.

In Lena’s case, cysitine in the eyes causes severe sensitivity to light and eyelid spasms meaning she must use eye drops up to once an hour to decrease pain levels and take medication which, while reducing the amount of amino acid, does not positively affect the eyes.

Now a team of researchers at RGU’s School of Pharmacy and Life Sciences have received a grant which will help them to develop an revolutionary new treatment for eye-related complications associated with cystinosis, which affects around 150 children in the UK.

The team has been studying the disease for five years and hopes the £101,554 SPARKS grant will help to create a gel treatment which will reduce pain and the intense dosing schedule and improve the sufferers’ quality of life.

Researcher Dr Graeme Kay said: “The difficulty with the current treatment of cystinosis is the unpleasant and time-consuming natures of the medicines themselves.

“For example, the oral treatment is very unpalatable and therefore the compliance of patients, especially children, has always been problematic.”

“Finding ways to get over this hurdle has been a core motivational factor for the team as it is only through a strict treatment regime that this debilitating condition can be controlled.”

Lena’s dad Roy, who is also chairman of Cystinosis Foundation UK, has welcomed the news, saying he and his family are delighted that a new treatment could soon be available.

He said: “Creating an eye gel that may only have to be used once a day will make a great difference to the lives of people and their families with this rare condition. We are always glad to support the high quality work carried out by the team at the university.”

Chief executive of Sparks John Shanley added: “The development of an eye gel which is intended not only to relieve pain but which should be less time consuming to apply, has huge potential for improving the treatment of cystinosis and the lives of the children living with this rare condition.

“As a charity we give careful consideration to the research we fund, so are delighted that we can give our support to this.”