The mum of a six-year-old boy who suffers from a fatal condition has thanked Echo readers for their support - despite the failure of what she hoped would be a lifeline drugs trial.
Ryhope schoolboy Jack Watson is not expected to live past his teenage years after being born with rare Sanfilippo disease, a degenerative condition which causes severe mental and physical deterioration.
Though the family has had to come to terms with the fact they will one day have to say goodbye to their little boy, a glimmer of hope came in the form of a drugs trial.
Three years ago, Wearsiders rallied round to raise around £18,000 to part fund the £600,000 trial at Manchester Children’s Hospital.
Jack was the first of 19 children nationally to take part in the on-going trial of Genistein. The drug has been shown to halt the progression of the disease in pre-clinical studies, but it needs to be tested in clinical trials before it can be prescribed in the United Kingdom.
Though the other children are still taking part in the trial, its effects were so detrimental to Sunningdale School pupil Jack that parents Gemma and Garry took the agonising decision to stop administering the drug.
“He changed so dramatically, it was heart-breaking to watch,” said mum-of-three Gemma, 33. “He was so spaced-out, which wasn’t like him at all, and he started crying when he went to school. Anyone who knows Jack knows that he would rather go to school than Disneyland.
“So we decided to stop the drugs, as we’d rather have him as his jolly little self, than the way he was on the drugs. The doctors at the hospital said we know our child best, so we could make that decision.
“We’ve also decided not to take part in anymore trials, to be given that hope and have it taken away from you is really difficult to come to terms with. But we’ve accepted it now.”
Fundraising events included charity football matches, raffles, gigs, head shaves and business donations which flooded in for Jack’s trial, but Gemma says their efforts have not been in vain.
“We needed to try,” said Gemma, who is also mum to Lewis, 13, and Lucy, three. “The trial may well still be a success, the results aren’t in yet, it’s just that it wasn’t for Jack. He was the first to start the treatment, but the other children are still taking part.
“He took the drugs for 18 months altogether. The doctors said one child would be given a placebo for the first year and we think that may have been Jack, as he was totally unchanged for the first year. It was in the six months after that that we noticed such a dramatic change in his behaviour and felt we had to make the decision not to continue. We didn’t want to keep going in case he changed any more.
“The drugs are supposed to halt the progress of the condition but, in Jack’s case, we felt he was deteriorating. Things he used to be able to do, like feed himself, he can’t do anymore. But that’s part of his condition as well. Though Jack looks like a six-year-old, mentally, he has the age of a nine-month-old baby.”
Gemma, who is married to oil worker Garry, says she can’t thank the people who donated to Jack’s fund enough.
“Words can’t describe how grateful we are,” she said. “All those people gave us the chance to save Jack and it may help children in the future. Because of all the publicity, people come up to chat to Jack in the Bridges and when we’re out and about. He used to be so wary of people, but it’s made him more sociable. People have got to know my hero, which is Jack, and it’s helped raise awareness of the condition too.
“It just goes to show how amazing people can be. And it’s happening again now with all the money being raised for Bradley Lowery.”
What is Sanfilippo Syndrome?
Jack, whose courage in dealing with the disease has earned him a Pride of Wearside award, is one of just 150 children in the UK today with Sanfilippo disease. It’s believed there are three children in the North East who have the condition.
Though Jack is no longer taking part in the drugs trial, he regularly attends the Niall Quinn Children’s Centre at Sunderland Royal where specialists monitor his condition.
Sanfilippo syndrome is a metabolism disorder in which the body cannot properly break down long chains of sugar molecules.
It’s an inherited condition, which means it is passed down through families.
Symptoms often appear after the first year of life. A decline in learning ability typically occurs between ages two and six. The child may have normal growth during the first few years, but final height is below average. Delayed development is followed by worsening mental status.
There is no specific treatment available for the condition, which is named after Sylvester Sanfilippo, the paediatrician who first described the disease.