'Game-changing': Desperate fight for drug to give Ethan, 11, longer life
For every extra candle added to 11-year-old Ethan Young's birthday cake, it is one more year the Norwich youngster is waiting for a breakthrough drug that could save his life.
Ethan, who like most young boys loves nothing more than playing computer game Fortnite, eating pizza, and trampolining, has cystic fibrosis (CF).
CF causes sticky mucus to build up in the lungs of sufferers, increasing their chances of serious lung infections and over time causing scarring that makes it harder to get oxygen.
The condition means sufferers have a capped life expectancy - the average life expectancy of someone living with the condition today is 40.
But, if a new precision medication manufactured by pharmaceutical firm Vertex is licensed in the UK - and crucially funded by the NHS - Ethan and around 10,000 others like him in the UK could live normal lives.
MORE: Plight of Norwich 11-year-old with cystic fibrosis cited by MP in drug pricing debate
Ethan's mother, 49-year-old Rachael Young, had to cut her working hours when she realised taking care of Ethan was going to have such an impact on the family's life.
She said: "We were told he had CF when he was four days old, he was in NICU [the neonatal intensive care unit] for two weeks.
"We had never heard of CF, we didn't know we had it in our family. We were completely devastated. I will never forget the consultant said 'most children make it to adulthood' which is quite a shocking thing to hear."
From then, CF had taken over the family's life, and Ms Young had been as realistic with her children as possible.
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Ethan's sister Emily, 13, learned about CF in biology at school and finding out the prognosis - which Mrs Young said was actually out of date - was extremely upsetting.
"I don't lie to them about it," Mrs Young said. "But it's not a conversation you want to have. The word Emily was told was it was progressive and Ethan said 'does that mean I'm going to get sicker?' and I said well it does, but it might not.
"I try to talk about the future and I know these drugs are coming and they are supposed to be game changing. That's what we have to hope for."
Trials are currently under way for triple combination therapy, using three of Vertex's drugs, to improve lung function.
Early results show there could be significant improvement in the conditions of 90pc of CF sufferers if they can access the medication.
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However, Vertex is also currently locked in a tense pricing battle with the NHS over another CF drug, Orkambi. It means that although the medication has been approved for use for more than three years, children and adults with CF in the UK cannot get it on the NHS.
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Campaigners say 249 people eligible for the drug have died in this time and around half of UK sufferers could benefit from it.
It is feared the same could happen with triple therapy.
Currently Ethan has two physiotherapy sessions daily, as well as taking a cocktail of drugs, and breathing in antibiotics and mucus thinners through a nebulisers four times a day.
But all of this only controls the symptoms of disease, whereas the new treatment could address the underlying cause.
MORE: Norfolk families affected by cystic fibrosis push for life-changing treatment to be made available amid price row
Theoretically, Ethan could start having the triple therapy when he turns 12 next year, if it has been licensed by then.
But if prices are too high for the NHS to pay - like with Orkambi - every year that passes Ethan will become more ill.
"If he's got to wait four or five years, his lungs are going to get worse," Ms Young said. "Potentially to the stage where it [the medication] then can't work."
Just recently Ethan's lung capacity had declined to just 54pc, forcing him to spend two full days in hospital in the past four weeks, plus enduring 14 days of intravenous antibiotics, which are given two times a day and take 45 minutes each time.
With a new treatment, Ms Young said it would cut down on hospital visits, and allow those with CF to live normal lives.
"Vertex has really been a point of hope for everyone," she said. "But there is no point in developing drugs that no one can then afford, so something needs to change or it's only going to get worse."
Ethan's plight was used as an example of why pharmaceutical companies should "put patients, not profits" first in parliament.
Speaking in a Westminster Hall debate last week Clive Lewis, Labour MP for Norwich South, said Ethan and his mother were "forced to live with the realities of cystic fibrosis every day, and the adverse impact that the actions of the company Vertex are having on their lives".
Ms Young said she had written to Mr Lewis and then spoken to him when he knocked on her door during campaigning for the European elections.
It was after that they met with the MP to see how he could help.
Despite the challenges, Ethan, an Avenues Junior School pupil, remains upbeat.
He said: "I'm not as worried because I think if you get worried about something it will make it feel worse, I try and stay positive."