Treatment trial could lengthen life of seriously ill Sunderland schoolboy

Jack Watson, four, who has been accepted on a life-changing drugs trial.
Jack Watson, four, who has been accepted on a life-changing drugs trial.
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A SUNDERLAND schoolboy with a rare life-threatening disease has been chosen to trial revolutionary treatment which could give him more precious years with his family.

For the past two years, Jack Watson’s family has been helping to raise funds to pay for a new study which could help prolong the life of the four-year-old who has the fatal Sanfilippo Disease.

Today it’s been announced that the hard work of the Ryhope family, their friends and kind-hearted Wearsiders who’ve donated to the cause to raise more than £18,000, has paid off.

This month Jack, a Sunningdale School pupil, will be the first child in the country to take part in a trial being run by researchers at The Royal Manchester Children’s Hospital and The University of Manchester.

Jack’s mum Gemma, 31, said: “If I could hang a banner off the Wearmouth Bridge to say thank you to everyone who has fund-raised, I would. I was skipping around the kitchen when I got the call from the hospital about Jack’s trial.”

The youngster’s story, which was first featured in the Echo two years ago, has touched the hearts of Wearsiders.

After reading about his plight, dozens threw themselves into fund-raising with events such as sponsored football matches, charity nights, a zip wire and more.

“People who didn’t even know Jack have done fund-raisers for him and he gets recognised in the streets now. People’s generosity has been amazing,” said Gemma, who is also mum to Lewis, 10, and Lucy, 13 months.

Jack, who lives in Ridgeway, is one of just 150 children to suffer from Sanfilippo in the UK, which causes hyperactivity, behavioural problems and progressive developmental delay.

The drugs trial aims to establish whether high doses of the treatment Genistein Aglycone is effective in people with the disease.

Gemma, who will marry fiancé Garry Watson, 34, this month, said: “The trial can’t save Jack’s life, but it can prolong his life, giving us more memories with him. We hope the research will help to save children with Jack’s condition in the future.”

Gemma found out about the trial through The MPS Society, which was working with researchers at The Royal Manchester Children’s Hospital. She teamed up with 19 other families across the UK to fund the trial, which will cost £600,000 in total.

Dr Brian Bigger, chief scientific investigator for the study from The University of Manchester, said: “It is fantastic to see a treatment we developed at The University of Manchester, in collaboration with clinicians, reach the point where we can test its effectiveness in patients. A US study published last year, demonstrated the safety of high doses of Genistein Alygone in patients with Sanfilippo Disease.

“This new study is the first to test the effectiveness of the higher doses of Genistein, which are thought to be of the strength required to see an effect in the brain. We are looking forward to analysing the results of the trial and hope that the treatment will benefit patients.”

People with Sanfilippo Disease have too much of a substance called heparan sulphate in their cells, particularly cells in the brain, because they lack the enzyme that usually breaks it down.

It is thought that Genistein Aglycone works by blocking the production of heparan sulphate and associated cell damage.