Cystic fibrosis: New drug gives Ivy, 6, 'a future'

Ivy loves horses but her condition limits how much she can be around them

A six-year old girl with cystic fibrosis finally has "a future" thanks to a new drug treatment, her mother has said.

Ivy Roberts is one of the first children younger than 12 in Wales to receive Kaftrio, a new "life-transforming" treatment.

"It's no longer a death sentence," said Kimberley Roberts, 33, from Dwygyfylchi, Conwy county.

"She can start living a better and more normal life, and live a longer life."

Ivy was diagnosed with the genetic condition when she was three weeks old following a heel-prick test which all new-born babies in the UK receive.

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Kimberley Roberts says her daughter Ivy will be able to live a longer life thanks to the new treatment

"It was a shock. We didn't really believe it," Ms Roberts said. "We sort of just grieved for the first year of Ivy's life."

From a very young age, Ivy has had to do physiotherapy two to three times a day as well as breathing exercises.

"Between two and four years old and then when she started school it was a constant battle. It made her really sad and miserable," Ms Roberts said.

Cystic fibrosis causes thick, sticky mucus to build up in the lungs, digestive system and other organs.

The condition gets worse over time and can cause breathing problems, infections and chronic lung disease. Patients may also struggle to gain weight and children can suffer from poor growth.

Ivy aged 4 with a viral cold. The nebuliser helps break down the mucus

Kaftrio combines three drugs: ivacaftor, tezacaftor and elexacaftor. It had already been approved for patients older than 12 in Wales in July 2020.

It has had a profound impact on the quality of life of some of the people benefiting from it.

In January, children aged six to 11 also became eligible for the treatment on the NHS.

'It's phenomenal'

"We've seen adults on the lung transplant list... come off the list. Cystic fibrosis sufferers struggle to gain weight and these people have gained weight within two weeks," Ms Roberts said of the new drug.

"It's phenomenal... it's made it treatable rather than a life-limiting disease."

Cystic fibrosis is caused by mutations in a gene. This gene contains the information needed to make a protein involved in regulating the production of mucus in the lungs and digestive system.

Kaftrio helps this protein to work better and helps to treat the underlying cause, rather than symptoms.

Ivy wearing her physio therapy vest which she uses twice daily for half an hour

She hopes to see a big improvement in her daughter's health in the next few weeks too.

Ivy takes 40 to 60 tablets a day to manage her condition and has about eight to 10 tablets with each meal. These include enzymes to help her digest food and vitamins.

"We became obsessed with cleaning. We didn't know how to handle the diagnosis," Ms Roberts said. "A simple cold could mean two weeks in hospital on intravenous antibiotics and as soon as she gets a sniffle at home she goes on antibiotics."

'We fought and fought'

By the time Ivy's first birthday approached, her parents had spent time researching the disease.

"I thought: I can either wallow in self pity or I can actively try to turn this around," Ms Roberts said.

They started campaigning for new drugs to be made available to younger patients and raised money.

"We fought and fought for this to be approved," Ms Roberts said.

She hopes parents with babies who are diagnosed with cystic fibrosis will now not face the same grieving process.

"The drugs are here."