A Blackpool family can see the “light at the end of the tunnel after 10 years of darkness” following Lancashire Teaching Hospitals NHS Foundation Trust’s decision to grant 13-year-old Tyler Palmer access to a pioneering treatment for Duchenne Muscular Dystrophy (DMD).
Tyler, who was diagnosed with the progressive muscle-wasting condition at age three, is now receiving Givinostat - a drug that has shown promising results in clinical trials for ambulant DMD patients, those who can still stand or take a few steps independently.
Givinostat is currently under review by the National Institute for Health and Care Excellence (NICE), with a decision on its wider NHS availability expected this autumn. All drugs provided through the NHS must undergo this rigorous process to ensure they are safe, effective, and offer value for money.
While NICE approval is pending, the drug’s manufacturer, ITF Pharma UK, has made Givinostat available to eligible patients through an Early Access Programme (EAP). Although the drug is provided free of charge, the programme does not cover the costs of safe delivery, including clinical monitoring and support - essential due to the drug’s significant side effects.
Lancashire Teaching Hospitals has worked to secure additional resources to provide the treatment to all eligible ambulant DMD children, with Tyler the first boy to be treated.
Though not a cure, Givinostat offers hope. It may extend mobility by several years and reduce complications such as the need for spinal surgery, potentially improving quality of life for boys with Duchenne.
Tyler’s father, Chris Palmer, shared the family’s journey: “Tyler was diagnosed when he was three. My wife noticed he wasn’t hitting milestones, and after many hospital appointments, we received the diagnosis. It’s been a 10-year battle. I reached out to Dr Christian De Goede, Tyler’s consultant, who has always had his best interests at heart. He’s a passionate advocate for neuromuscular conditions.
“In February, I emailed him about Givinostat. Through persistence - from myself, Mr Steve Canty - Divisional Medical Director and incoming Chief Medical Officer, Dr De Goede, and support from a small group of Preston parents - we pushed until funding was secured. We wanted to be that ‘thorn in the side’ that opened the door for other families too.”
Chris added: “Tyler currently has two months’ supply of the drug. One of the side effects is low platelet counts, so he’ll have blood tests every two weeks for three months while they monitor and adjust his dosage. The hope is that it will slow progression, improve quality of life, and even promote new muscle growth. Duchenne is a degenerative, terminal condition - but this drug offers hope. It changes the curve a bit. It buys time.
“For us, after 10 years of darkness, this feels like a bit of light at the end of the tunnel. Watching someone you love go through this is unbearable - you’d swap places if you could. But this drug gives us a little bit of hope for the future.”
And Chris thanked the backing of a number of parents and MPs: "While Tyler’s story is one part of it, none of this would have been possible without the dedication and contributions of other parents at Preston, including Bernie Mooney, Susi Hardman, Andrew Hargreaves, Ashley Steele, and Leanne Witts.
"I also feel it’s important to acknowledge the support of local MPs - Chris Webb, Sir Lindsay Hoyle, Lizzie Collinge and Maya Ellis, as well as the vital role played by Alex Johnson and Katie Combes of Duchenne UK, and Muscular Dystrophy UK."
Mr Canty said: “We are pleased to be able to offer Givinostat to Tyler and all eligible ambulant DMD children through the Early Access Programme and provide some hope to those families."
