A 12-year-old Newry boy living with Duchenne Muscular Dystrophy (DMD) has had the opportunity to meet with Northern Ireland’s Health Minister to discuss access to a new trial drug which has so far remained out of his reach.
Alfie Pentony has – since his diagnosis in 2016 – fought with the help of his parents Jamie and Colleen for access to the best possible treatments to help slow progression of the inherited muscle wasting condition.
For the last number of years, Alfie has been attending bi-annual appointments at a private hospital in Newcastle, England alongside taking steroid medication to slow the degenerative effects of his condition, interspersed with yearly visits to America for advanced specialist treatment – all coming at a considerable cost to his family.
In February of this year, however, Alfie’s mum Colleen was made aware of a new “life-changing” medication that had been approved for UK use by Medicines and Healthcare products Regulatory Agency (MHRA) for all people with DMD aged six and older.
Known as Givinostat, Duchenne UK – who had been campaigning for its use since April 2024 – described MHRA’s approval of the drug as “a very special day”, writing to 260 families to inform of its availability through an ‘Early Access Programme (EAP)’.
The drug had been proven to assist with slowing the condition, allowing sufferers the ability to remain independent and mobile for an extended period of time.
However, Colleen spoke to Armagh I in February about her concerns that Alfie and 12 other Northern Irish boys living with the condition were unable to participate in the EAP – due to the cost to The Health Trust and potential storage issues.
In a letter to Colleen from the Health Minister, Mike Nesbitt in January, he explained the reason for delayed access to the drug, saying: “Access to the EAP for Givinostat is subject to meeting specific criteria set by the manufacturer.
“If access is approved by the manufacturer, the medicine will be supplied to the Trust at no cost. However, the Trust will be responsible for any additional costs associated with treatment administration.”
Left to right: NI Health Minister Mike Nesbitt with MLA Liz Kimmins, Colleen and Jamie Pentony and Alfie Pentony (front).
Both Jamie and Colleen regarded the delay as a “failing” – especially given that they were watching Trusts in England moving at a much quicker pace.
And, “time”, says Colleen “is muscle” for people living with DMD. A criteria to be considered for admittance onto the Givinostat EAP was a need to prove “ambulatory” which is often an unknown for someone with such a severe muscle wasting condition.
In the last two months, there has been a semblance of progression, however, as Alfie – along with another 11-year-old boy – met with Northern Ireland’s Health Minister Mike Nesbitt to address their concerns, together with their parents and extended families.
While Colleen says the Belfast Health and Social Care Trust is still “not going to provide access” to the EAP for Givinostat, the opportunity to meet with the Health Minister has been taken as a positive step forward.
Said Colleen: “We raised concerns about numerous issues, and every parent agreed that the neuromuscular service under Belfast Health and Social Care Trust is failing to deliver the proper standard of care, let alone provide early access to Givinostat through the EAP.
“And of course Alfie again told the Minister he just wants other boys to get the drug. Too many of his friends have already came off their feet and it’s breaking his heart.
“Minister Nesbitt took the time to listen to our boys’ and families’ stories and experiences with the Trust.
“It is now clear to him that systemic issues exist within the neuromuscular services for boys with Duchenne Muscular Dystrophy (DMD).
“We left the meeting hopeful, based on what he heard, that he will urgently address these problems and adopt a positive approach to help our children. Their lives matter.”
Colleen also thanked her “good friend” and local Sinn Féin MLA Liz Kimmins and Sinn Féin MLA Linda Dillon for organising the meeting.
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