23-04-2025
Children denied access to life-changing drug is 'unfair' and 'frustrating' say families
ITV Granada Reports correspondent Mel Barham speaks with two families campaigning for better access to a 'life-changing' drug.
The families of children with a severe genetic muscle-wasting condition say it's 'unfair' and 'completely frustrating' that they're being denied access to free drugs that will slow its progression.
Givinostat is a drug that helps treat patients with Duchenne Muscular Dystrophy.
17 year old Jack Johnson has been on the drug for the last five years as part of a clinical trial and says it's made a massive difference to his life: "It makes me feel really sad because I want to help every boy with Duchenne, I want them to have the same opportunities I have been given with this drug."
Granada Reports first spoke to Jack Johnson over a decade ago, when the famous 'Joining Jack salute' was adopted by sports stars and celebrities, helping the charity raise millions of pounds for research into the condition.
They helped fund a clinical trial for a new drug called Givinostat, which was found to be effective at slowing down the disease.
But while Jack's been given access to the drug, only six NHS trusts in the country and none in the North West will treat patients with it.
That's despite it being approved as a licensed drug in November and the pharmaceutical company offering it for free to the NHS as part of an early access means there are many boys with Duchenne across the country being denied access to a treatment that could potentially help them.
Jack's mum, Alex Johnson says: "I'm completely frustrated, I can't believe it. Everything my family and everyone has done in the North West over past 13 years to get this clinical trial up and running and then to get the drug approved... for us now to be in this situation where free medication cannot be given to these boys, is disgusting."One of those, is nine year old Jack Binns. He's been told because of where he lives in Cheshire, he can't get Givinostat.
His mum, Jill Binns, says: "It makes me very sad and very angry. It's just so unfair. We're constantly battling. We just want to see them have a prolonged life and he doesn't understand."Jack Johnson says: "It doesn't make any sense why the NHS wouldn't want to help, that's what they're there for, to help people and this medicine is going to do that."
Alex says: "At the minute it just feels like the patients are pawns in a big chess game and they are the ones that are suffering."
"I really would encourage the management of these trusts to actually remember that these are boys that deserve this medication, they deserve to try a treatment that is going to slow down the progression of this disease for them."
The National Institute for Health and Social Care will assess the drug for cost effectiveness in they approve it, it would be made available across the NHS.
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD) is the most common and severe form of muscular dystrophy.
It causes muscle degeneration and weakness due to a mutation in the gene that produces dystrophin, a protein that protects muscle fibres from breaking down.
The condition usually only affects boys, with many patients unable to walk by the age of 12 and usually only surviving into their 20s and 30s.
There are about 2,500 people in the UK living with DMD at any one time, with about 100 new diagnoses each year.
