'This drug changes everything': Family's relief as little Amélie today begins new 'life-saving' cystic fibrosis medication, Kaftrio

After years of campaigning by people with the degenerative condition and their families, the miracle drug, Kaftrio, has finally been approved for children aged six to 11 - and today Amélie will begin her treatment.

Her emotional mum, Natasha McIntyre, told the Yorkshire Evening Post: “I cried when they [the drugs] arrived.

“It was just an enormous sense of relief. Something that we have been working towards ever since she was diagnosed. It’s amazing.”

Cystic fibrosis, an incurable condition caused by a faulty gene, leads to a build-up of sticky mucus in the lungs and digestive system, making it hard to breathe and digest food.

In 2019, of those who died from the condition, the average age was just 31.

But experts believe this innovative drug - which until recently was only licensed for those aged over 12 - is an important step in limiting lung damage and slowing long-term decline in health and has been hailed as “life-saving” by the charity Cystic Fibrosis Trust.

Kaftrio attacks the underlying causes of the disease as well as the symptoms and Amelie is among an estimated 1,500 children across the UK and Northern Ireland who will benefit - limiting the damage CF does in the critical early years.

Natasha said: “In theory it should mean she has a normal life expectancy. It should give her the same life chances as any other child born at the same time as her.

“It would mean that she has less chest infections. But even if she does get a chest infection, less damage is done. Better vitamin levels, better growth.

“It will mean that the cells in her body are working normally.”

Currently Amélie’s daily treatment regime involves a cocktail of drugs to ward off infections, including Creon with every meal to help her digest food, as well as daily physio - where someone pounds on her chest to clear mucus in her lungs.

While Amélie hopes this will change due to the drug, Natasha says that’s not yet been the case for those she has talked to who are already on it - but still the benefits are clear.

She said: “It’s just that improvement in quality of life that they are experiencing.

“I know of a man whose brother has CF and he was allowed to start taking it early because his lung function was low - it was at 14 per cent. His lung function [per cent] is now in the 70s. They weren’t expecting him to live much longer and now he’s fine, and he’s had a baby.

“It’s essentially saved his life and to think that that is available for my daughter and lots of other people now.

“It will give her more life chances.”

Natasha said this drug has lifted the dread of having to have a conversation with Amélie further down the line, about what her condition might mean for her future.

“It means that we don’t have to have that conversion as early. We have not got to talk to her about the fact that she might die before she’s my age because this drug has changed everything. I wasn’t looking forward to having that conversation with my child.”

And while Amélie doesn’t understand the full enormity of the drug, she is aware it will change her life.

“She has started to become aware of the limits of her condition. But in theory it should be a safer world for her and she’s a bit happier now that we have had it delivered.

“She knows it’s not a cure but she knows it will keep her feeling well for longer and she won’t cough as much. She won’t get so out of breath when she is exercising.

“She knows that she should find it easier to breathe.”

Natasha added: “I just can’t wait for Saturday, I can’t wait for her to start this drug. It’s like all our birthdays have come at once.”

Amélie’s grandma Julie Faulkes, 66, also spoke of her relief and hope for the future.

“When she was born, we were very distressed by her diagnosis but since then, over the last six years, there have been so many advances and we are just relieved that she is now getting Kaftrio, because it means that she will give her a near-normal life expectancy.”

She recalled a childhood friend of Natasha’s who had CF but sadly died when she was 22.

“That’s always been at the back of our minds," she said. "That we might not have Amélie for too long. But this gives you so much hope.

“She’s such a lovely little girl and my daughter and son-in-law have coped really well with her condition."

It will be an emotional day for the family as Saturday also marks the anniversary of the death of Amélie’s other grandmother, Angeline McIntyre, who died three years ago aged 73.

Natasha said Amélie getting access to drugs like Kaftrio was something Angeline had been desperate for.

“It would have absolutely made her year to hear that Amélie could finally have this drug," she said.

*Family friends of Amélie are currently raising money for the Cystic Fibrosis Trust.

South Leeds pub, the Greyhound in Birstall, is holding a pool tournament from April 30 to May 1, with a raffle and donation buckets to raise money for the charity.

And family friend Oliver Harrison, 39, is aiming to run 100 miles in 24 hours for the Cystic Fibrosis Trust in July at Endure24, a 24-hour running race at Bramham Park. To sponsor him, visit https://justgiving.com/fundraising/oliver-harrison16.

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