Girl, 7, with cystic fibrosis has 'bright future' after receiving 'revolutionary' drug on NHS

ITV News Northern Reporter Sangita Lal meets Kate Farrer who is hoping for a 'fairer' life on the new drug

A seven-year-old girl with cystic fibrosis said she hopes "life will be a bit fairer on me" after being among the first of hundreds of children to receive a "revolutionary" treatment for the disease on the NHS.

More than 1,300 children with the disease in England are newly eligible for drug Kaftrio, after it was extended for licence by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

The drug was first approved for those aged 12 and above with cystic fibrosis - which causes sticky mucus to build up in the lungs and digestive system - in England in 2020. But last month the treatment was approved for children aged six to 11.

It means children with the disease can start receiving the treatment, which tackles the underlying causes of the disease as well as symptoms, within weeks.

Kate Farrer, seven, started taking the drug on Sunday and was "really excited" that it began to take effect within three hours.

Kate Farrer said "it's a life-changing drug and it's going to help me a lot for when I'm older"

The little girl has spent much of her life in and out of hospital, particularly over the past couple of years, having suffered from pneumonia and other difficult conditions.

Katie told ITV News: "A lot of mucus has been coming up and I'm feeling a lot better now.

"It was really fun getting it because now life will probably, or hopefully, be a bit fairer on me because sometimes it's not as fair because of all of my treatment and medication."

Kate has spent much of her life in and out of hospital with the disease Credit: Supplied

Kate, who loves sports and gymnastics, said her illness has meant she has had to miss out on many things like school trips, while her mum Catherine Farrer said she is struggling to keep up with friends.

The schoolgirl currently spends up to an hour-and-a-half a day doing physiotherapy, which involves blowing hundreds of times into different machines, while her parents have to be prepared with medication at all times for any complications.

Mum Catherine, who has been campaigning for years to get to this point, told how being able to give her daughter the treatment on the NHS was an "enormous relief".

"I can't describe what it's like to be told your child has got a chronic condition"

She told ITV News: "It's been tough. I can't describe what it's like to be told your child has got a chronic condition. It was devastating and we obviously feared for her future."

"She is getting worse, it is becoming more of a burden, we are needing more treatment, more antibiotics... more hospital admissions," Catherine added.

The mother said it's been "quite the battle" to get hold of the medication but the results so far have been "amazing", with Kate coughing up mucus and clearing her lungs within hours.

She said she can't wait to see more children access the drug and that her daughter's future is now "really bright".

"Our hopes now with this treatment is that things should start to get easier not just for us, but for Kate as well, she will have more time to live and be a seven-year-old," she added.

Mum Catherine explains the impact the drug Kaftrio had on her daughter within hours:

What is cystic fibrosis (CF)?

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits people’s ability to breathe over time.

There are around 8,000 people living with CF in England, which is the second highest proportion in the world. There is no cure for CF but patients need to take different medicines to treat and prevent lung problems.

According to the NS, symptoms of cystic fibrosis include:

  • recurring chest infections

  • wheezing, coughing, shortness of breath and damage to the airways (bronchiectasis)

  • difficulty putting on weight and growing

  • yellowing of the skin and the whites of the eyes (jaundice)

  • stomach pain including diarrhoea and constipation

  • a bowel obstruction in newborn babies, for which surgery may be needed

People with CF have a higher risk of developing other conditions such as diabetes, liver problems, fertility issues and osteoporosis (weak and brittle bones), and are more vulnerable to complications if they develop an infection.

How could Kaftrio help CF patients?

Kaftrio is a triple combination treatment that tackles the underlying causes of the disease by helping the lungs work effectively.

It helps to make patients' lungs clear and significantly improves lung function, helping people with cystic fibrosis to breathe more easily and enhancing their overall quality of life.

Dr Cara Bossley, a paediatric respiratory consultant at King's College Hospital, sets out how the drug will work:

The treatment combines three drugs which perform different functions – ivacaftor, tezacaftor and elexacaftor.

It needs to be taken twice a day for the rest of patients' lives in order to be effective but if they start the drug early enough, they will live "healthy long lives," said Dr Cara Bossley, a paediatric respiratory consultant at Kings College.

"We know that starting this treatment early in life can be absolutely life-changing and stop any irreversible damage on the lunges of children with cystic fibrosis," she told ITV News.

Dr Bossley said the main draw back to the drug is that medics have to monitor liver function through regular blood tests, as the medication goes through the liver - but "most patients tolerate the drug really well".

She said medics are very "excited" about the rollout as they believe it will improve patients' day-to-day lives but also extend their lifespan.

In time they hope to roll it out to even younger children.

For Kate, she said she's now looking forward to having less tummy aches and enjoying more school trips and gymnastics practices without coughing.

She said: "I'm really happy because it's a life-changing drug and it's going to help me a lot for when I'm older."