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Twelve year old Newry boy Alfie Pentony was part of a heartfelt discussion on the Stephen Nolan Show earlier this week about the lack of availability of a new drug that could help treat his Duchenne Muscular Dystrophy.
Alfie who spoke on the show along with his father Jamie, was diagnosed with the life limiting condition in November 2016. A new drug, Givinostat, could help him and others walk longer, but it's only available in England. The Belfast Trust, responsible for people with the condition in Northern Ireland, declined to apply for the drug due to capacity issues. The drug, which requires only seven blood tests over six months, could significantly improve Alfie's and other children's lives. The Department of Health has acknowledged the issue but cites regulatory and resource constraints.
Following the radio interview Alfie's plight was also brought up in the Assembly on Tuesday.
There are around 50 boys in Northern Ireland with Duchenne's but out of this number Alfie and another 12 of the boys are the only ones eligible for treatment by the new drug as it's not for people under six and not for people who can't walk.
His father is worried that by the time the drug does get issued that it may be too late for some. Alfie has got stem cell treatment in America over the past few years and it has helped him a lot meaning that he should still be walking this time next year, but others are not so lucky.
"I personally know kids that aren't going to be walking this time next year." said Jamie.
"We need some goodwill from the Trust. We need somebody to shuffle the deck, move some things around so we can treat him."
Despite his youth Alfie was incredibly on-message during his emotional interview. "My life expectancy is not as much as everybody else, and I could be in a wheelchair, but luckily, I'm not." Alfie said.
"It would really help me, it would help me a lot. But there's other children that are going to stop walking before me, so it would be important for them to get it as well."
He added "It's cruel that they're not giving this drug to kids in Northern Ireland with this disability when most of them won't be walking next year. It's just horrible. I think we all have the right to have something that could help but they just have said they don't have the staff to do this. It's not good at all."
Jamie said "We're standing up to make sure that his wee voice isn't getting ignored, and the other 12 boys and the other parents aren't getting ignored, and just shunned away off and go we can't do it, and that's it. This is the first thing that's come out in 20 years that could help my son."
'Cruel' lack of access to drug discussed at Assembly
At the Assembly on Tuesday, South Down MLA Colin McGrath asked the Minister of Health,Mike Nesbitt whether it is fair that Alfie and others are being deprived of access to such drugs.
Minister Nesbitt said: It is my ambition that there be a UK-wide facility that would bring on any drug. NICE and the Medicines and Healthcare products Regulatory Agency give us advice, and that is the advice that we follow.
"In this instance, it is my understanding that the Belfast Trust makes the decision on whether this young man is allowed that drug. I am following up with the Belfast Trust to further understand its decision-making process. Several Members have approached me about this new drug for their constituents. Although I am not a clinician, on the face of it, it looks as though it is a revolutionary and very positive development, and I am keen to see it rolled out at pace, particularly for a young man such as the one whom you referenced."
Mr McGrath added: "I reiterate the point about moving "at pace", because timing is critical. If the people involved do not get access to the drug before the start of muscle wastage, the drug is of no use to them. The word that Alfie used today about not getting access to the drug was "cruel". That is the word of a 12-year-old to Members of this Assembly. It is cruel, and I hope that we can overcome and ensure that he, along with others, is able to get access to those drugs."
Minister Nesbitt assured him that "I am on that case"
