£600k needed for Fairford teenager to trial 'wonder' drug (From Wilts and Gloucestershire Standard)

£600k needed for Fairford teenager to trial 'wonder' drug

2:00pm Wednesday 12th September 2012 in News By Laura Shack

A FAIRFORD teenager who has a rare terminal disease could be given a "last chance" to live with a new revolutionary drug trial, if the £600,000 needed to fund it is secured.

Ollie Robinson was six-years-old when his parents first discovered his hyperactivity and challenging behaviour were symptoms of Sanfilippo, an incurable rare form of a degenerative disease called Mucopolysaccharide (MPS III).

Now at 13 years, Ollie has the mental capacity of an 18-month-old and has already reached his expected lifespan.

But soon, he could be one of 20 children with Sanfilippo to trial a new soy-based drug called Genistein aglycone, which could reduce hyperactivity in sufferers, slow down damaging toxins and stop the disease in its tracks completely.

Mum Karen has fought for years for research into the disease that affects around 130 children in the UK and, with the help of friends, has raised thousands of pounds for the MPS Society.

Karen said: "It’s incredibly exciting and I never dreamt that it would be available in Ollie’s lifetime. Potentially if the drug stops the toxins from building up in his brain he could live so much longer.

"The not knowing is the worst part. Anything that can buy us time and give him a chance of life is worth doing. And this is really a last chance option for us."

Before the relatively cheap drug can be prescribed for children with the disease it must be assessed in a controlled clinical trial at Manchester Children’s Hospital, which will cost hundreds of thousands of pounds.

All the early studies at the University of Manchester have been funded by the MPS Society, but a further £600,000 must be found before the first child can be treated.

Karen is hopeful that medical advances will not only find a cure but also raise awareness of the rare disease and its impact of affected families.

She said: "We call Ollie the master of mess because he’s always causing mischief. He can do very naughty things and people don’t understand why. "It can be very socially isolating because of his behaviour. But for us it’s made us better people – more positive, tolerant and strong as a family."

To find out more about MPS go to mpssociety.co.uk or to make a donation towards the Genistein clinical trial fund visit shareagift.com/jack