Scots butterfly girl's 'life changed' as miracle drug approved for painful condition

A miracle new treatment approved yesterday is set to change the life of Butterfly girl Isla Grist who suffers in agony every day.

Scotland legend Graeme Souness' wee pal Isla, 15, has the agonising condition Epidermolysis Bullosa – which causes the skin to blister and tear at the slightest touch. In January Souness - who is close pals with Isla and her family - begged health chiefs to help her by fast-tracking a new treatment which could finally ease her pain.

And yesterday their wish came true when the Scottish Medicines Consortium approved the use of Filsuvez gel, as the first licensed treatment in Europe to treat the devastating condition. Filsuvez is a sterile gel for wounds associated with EB in patients six months and older.

It was approved via the SMC’s ultra orphan medicines pathway for an initial three-year-period whilst further safety and efficacy data is collected. Graeme, befriended Isla and her family while fundraising alongside her dad, Andy, for the condition.

Along with a team of Royal Marines, Graeme and Andy swam the English Channel last year to raise awareness of the condition and raise funds for vital research into better treatments.

And a new challenge by Graeme, Andy and the team is expected to be announced later this month.

Isla has called them her “heroes” and she recently said: “Graeme is kind, very kind. He has a bit of a hard shell but he is squishy inside.”

Isla’s dad Andy, from Inverness, had also called on the SMC to make a speedy decision saying six months ago: “Any topical cream or liquid that helps skin heal quicker and stronger would have a massive effect.

“I have seen the data from trials and it is extremely positive. We are very excited.”

Isla’s skin is permanently covered in bandages because her skin below them is blistered and bleeding.

On average she is in hospital every two or three months. She has had more than 60 general anaesthetics for operations including those to stretch her throat so she can swallow food because the condition does not just affect external skin.

She has also had 15 operations on her hands - among the most painful of her many surgeries - because her fingers curl together.

Three times a week the Pride of Scotland Award winner has her dressings changed and has creams used to treat burn victims applied to the open weeping wounds which cover her little body.

The procedure can take up to five hours. And Isla admitted: “It hurts when the cream is applied.

“I don’t know if there is any way to describe the pain I am always in. It is at a high level. It is constant. It’s itchy. It tingles. Sometimes your body feels as if it is on fire.”

The Filsuvez gel treatment, made from birch bark extract, has had impressive results with many patients on a trial having full wound closure in 45 days and reduced pain when the dressings are changed. The body surface area covered in wounds also reduces by half in some patients.

Patients, like Isla, with dystrophic EB suffer constant pain from open wounds and are at an increased risk of vision loss, scarring and skin cancer. Most patients rarely survive beyond age 30.

Tony Byrne, boss of EB Charity Debra, said: “People living with all types of EB desperately need treatments that can positively impact their symptoms and improve their overall quality of life, so it is hugely encouraging that the first treatment for patients with junctional and dystrophic EB was recommended by NICE in September last year and has now been recommended by the SMC, making the treatment available across the UK.

“On behalf of the EB community, I would like to thank Chiesi and the SMC for facilitating this approval, and all our members who supported the application. “There is still much work to do to ensure approved treatments are available for all types of EB but this is a promising first step.

“It provides hope today and will hopefully be a catalyst for future therapies.”

Peter Marinkovich, MD, director of Stanford Medicine’s Blistering Disease Clinic said with Filsuvez: “The wounds heal quickly but, more importantly, they stay closed.”

Commenting on the approval, CEO at DEBRA (Dystrophic Epidermolysis Bullosa Research Association) UK, Tony Byrne, said the announcement was “hugely encouraging” for the EB community, while Head of Rare Diseases at drug company Chiesi UK&I, David Garzón Lafuente, emphasised the “deep commitment” they have to supporting people living with a rare disease.

The gel should be made available to eligible patients by September.

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