New drugs-trials hope for Sunderland youngster Pascal

I'm on the run, son: Patrick Bailly is doing the Great North Run  in aid of Muscular Dystrophy, which afflicts his son, Pascal.
I'm on the run, son: Patrick Bailly is doing the Great North Run in aid of Muscular Dystrophy, which afflicts his son, Pascal.
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SCHOOLBOY Pascal Bailly is today hoping his birthday wish comes true as he prepares to join a register for pioneering muscular dystrophy drugs trials.

The youngster, who was nine yesterday, was diagnosed with the Duchenne form of the disease five years ago.

The incurable condition gradually weakens the muscles around the lungs and pelvic area.

By the time Pascal is 10, he will probably be confined to a wheelchair.

His family have welcomed the news that research has shown for the first time it is possible to counteract the effects of the illness throughout the body.

Now his father Patrick, 45, from Nookside, Sunderland, plans to register his son for the next round of drugs trials.

“We’re sorting out the paperwork now, filling in the forms, to let them know we are available to take part in the trials,” said the caretaker. “Hopefully, we will be successful.”

Pascal, who attends Grindon Hall Christian School, is facing a race against time to treat his condition.

“The drugs will only slow down the effects of the condition. It is not a cure, but we hope they will allow him to get by without a wheelchair for much longer,” said Patrick. “But once he is wheelchair-bound, we won’t be able to reverse that.

“They will only help ease his heart, respiratory and spinal problems.”

Children recently took part in clinical trials at Newcastle’s Royal Victoria Infirmary and Great Ormond Street Hospital in London.

Seven out of 19 patients aged five to 15 responded to the injected drug in an early-stage clinical trial.

Professor Kate Bushby, from Newcastle University, said: “This drug actually targets the specific faults in the gene that some people with Duchenne muscular dystrophy have so that the gene can be, in a way, fooled into making a more functional protein that we believe will protect the muscle better in the long run.

“We’re not so far along the line yet that we can say that it stops the progression or reverses the cause of the disease because this was an early-stage study, but what we have shown is that the drug was very safe and very well tolerated.”  

Prof Bushby added: “We have to be realistic about this – it is not a cure but it certainly is the first time we’ve been able to really look at changing the protein level in the muscle in a way that we believe will be realistic in terms of providing some change in the condition.”