A breakthrough new drug, which could be available in England within six months, must be made available here before it is too late for a little boy from Limavady.
The little Limavady lad, four-year-old Callum McCorriston, suffers from a rare and acute form of muscular dystrophy, known as Duchenne. His incurable condition will cause a steady and severe deterioration in his muscles, leaving him wheelchair bound by the time he attends secondary school and paralysed by his late teens. He is not expected to live beyond his twenties.
One ray of hope for little Callum has just emerged, though, with a green light from the European Medicines Agency for the first drug in history to successfully treat Duchenne muscular dystrophy.
The new drug could be available on the NHS in England in just six months, although a campaign group has warned it could take as long as 18 months.
The drug works only for a minority of Duchenne sufferers - those whose condition is caused by what is known as a ‘nonsense’ genetic mutation. Callum is amongst that small group of children who could benefit from the revolutionary new treatment.
However, his mother, Laura, has warned that there is no guarantee it will be freely available on the NHS here in Northern Ireland.
“The NHS in England and the NHS in Northern Ireland are two different things”, she said. “The Muscular Dystrophy Campaign say it should be available within 18 months. It could take as long as that for it to be available on the NHS in England. But Callum doesn’t live in England, he lives in Northern Ireland. ”
She added: “At some stage over the next 18 months someone or some group of people are going to have to make a decision about whether the drug should be available in Northern Ireland.
“My major concern is that we know of only two boys that this drug will possibly help – and one of them is Callum.
“It really is quite simple. Just give us the medicine – get it approved, get it available, get it over here and give it to the boys. It sounds so easy. Unfortunately it doesn’t work out like that.
“My fear is them saying ‘sure it is only two boys.’ The question they will ask is ‘do two boys matter?’ That’ll be the part where we stand up and say ‘yes, they do.’ It is us standing up and saying ‘this is our son and he does matter.’”
Laura warns that time is running out before the groundbreaking new drug can no longer help her little boy.
“It worries me that this medicine has been approved for boys aged over five but who are still ambulant, still able to walk”, she said.
“If it is going to take 18 months for this to become available on the NHS in England, I don’t know what is going to happen in that 18 months.
“No-one can say to us: ‘He is definitely going to be walking in 18 months’. If they do take their time about it, he could be in a wheelchair by then and he won’t be given the drug. It is a race against time.
“For me, there is an element of excitement about it but at the same time there is so much apprehension, there is so much uncertainty and so many unanswered questions.
“I don’t know if we’re going to get it in Northern Ireland. I don’t know if Callum will still be on his feet when, and if, it becomes available here.”#
DUCHENNE MUSCULAR DYSTROPHY
Callum’s illness is a particularly rare and debilitating form of Muscular Dystrophy, known as Duchenne. It will radically reduce his life-span. There is no cure.
After decades of research, a new drug has been developed to treat the condition, known as Translarna or ataluren. The medicine prompts the body to manufacture a protein that protects against muscle damage which is absent in children with the disease.
The European Medicines Agency has recently recommended the drug for approval under special measures. It is the only treatment available for sufferers of Duchenne with evidence of effectiveness. It is to be granted a licence as an ‘orphan drug’ for rare diseases.
APPROVAL AND RESPONSE
The decision by the European Medicines Agency has been hailed as “fantastic news” by Robert Meadowcroft of the Muscular Dystrophy Campaign in Britain. “Most of those diagnosed with Duchenne muscular dystrophy, usually before the age of five, will use a powered wheelchair before they are 12, will not have the muscle strength to pick up a glass of water by the age of 20 and will not live to see their 30th birthday.”