‘Please don’t let us die’: Three cystic fibrosis sufferers beg the NHS to fund ‘wonder drug’

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It’s been hailed as a wonder drug with the potential to extend the lives of thousands of people with cystic fibrosis.   

But patients across the UK still can’t get their hands on Orkambi – despite it being a year since Theresa May promised to take action.

The drug, which slows lung deterioration, received its European licence three-and-a-half years ago, in which time over 200 people have died from the cruel condition. 

However, the National Institute of Health and Care Excellence (NICE) has been unable to reach an agreement with its manufacturer.

The Goverment-run body and NHS England have been in a deadlock with US firm Vertex over the drug, which costs an estimated £104,000 per patient per year. 

On the anniversary of the Prime Minister’s pledge on May 16, furious campaigners marched to 10 Downing Street to share their frustrations.

Mrs May responded last night by telling the firms to do ‘everything they can’ to break the deadlock. 

And MailOnline has spoken to three cystic fibrosis sufferers, who have begged the parties to make headway.

Hannah Chew née Lindley, 23, Shiloh Howells, nine, and Lorcán Maguire, two, are desperate for the chance to try Orkambi. 

Mrs May said she wanted a ‘speedy resolution’ in the ongoing negotiations so CF patients could have access to Orkambi and Symkevi –  another drug that treats the root cause of the condition but is also not available on the NHS.

Since then, there have been no resolutions.

Vertex reached an agreement with Scotland for access to Orkambi and Symkevi in December, but the fight for the drug is still raging on in England, Wales and Northern Ireland. 

Yesterday, the Cystic Fibrosis Trust handed a letter to Theresa May at number 10 Downing Street.

Zoey Jones, the distressed mother of one-year-old Eve, who has cystic fibrosis, was also there to give 65 roses – a term used by children to help them pronounce the name of the condition. 

David Ramsden, chief executive of the Cystic Fibrosis Trust, said: ‘This tragic situation cannot continue. Every day the health of thousands of people in this country is deteriorating – and this is damage to their lungs that can’t be undone.’

‘We’re asking for Prime Minister, Theresa May to attend the Westminster Hall debate on Monday 10 June and update the house on what the next steps are.’     

Hannah Chew née Lindley, 23, said CF has stolen her joy, childhood and her best friend – she tragically had to watch her sister, Iona Kesteven, pass away from the condition.

Hannah, of Leeds, isn’t afraid to admit that her life with CF, apart from some happy memories, has been ‘horrific’.

She said: ‘A lot of people mask it over which annoys me because it needs to take it seriously.

‘It’s stolen my joy, health and mobility. It stole my childhood and my best friend – my sister. I had to watch her die.

‘There are some days you feel better and there are others you question why you are on this planet.’

When Hannah looks back on her childhood, she mostly remembers Iona, who died at the age of 19, being ill.

She said: ‘We lived life permanently in a touch and go situation. I remember my sister being taken ill all the time and not knowing if she was going to live or die and constantly living with the fear of – is this the time she dies?’

Hannah’s parents tried to make their lives as normal as possible, taking the girls to ballet, piano lessons and performing groups.

She said: ‘We even went to the theatre to perform from the hospital. We did our makeup in the hospital. We didn’t want to be another number of kids stuck on the ward. We minimalised being in there as much as possible.’

But around the age of eight, Hannah remembers the illness taking a tighter hold of Iona, and they were put in separate bedrooms.

People with CF grow bugs in their lungs which are usually harmless to people who don’t have the condition, but can be easily transmitted from one person with CF to another, including siblings, and be very harmful. 

Hannah said: ‘I felt a lot of guilt because I wasn’t as poorly as she was.’

Iona was known to her consultants as being a complex patient, but Hannah believes it was May 2015, when Iona picked up a stomach bug on the flight home from a holiday, that things went severely downhill.

In November 2015, her lung collapsed due to multiple lung infections and she was rushed back into hospital. A week later she passed away.

Hannah, who married Nathan Chew, 23, in August 2018, said: ‘One of the consultants was in shock because they believed she would have survived longer. 

‘She could still be here with Orkambi. My sister died waiting for that drug. It was approved on compassionate grounds two weeks after her death but it was too late.

‘The healthier you are, the better chance you have, so they shouldn’t be waiting for them to get to a certain point.’

Hannah, who is finishing exams to become a music teacher, has won awards with the Cystic Fibrosis Holiday Fund for her ambassador work. She gained more than 23,000 signatures on a now closed petition. 

She said she is frustrated that CF patients with other mutations are given drugs that can treat them – despite being happy for them.

She said: ‘It’s disgusting. It’s like saying we will treat breast cancer but not lung cancer. Or type 1 diabetes but not type 2.’ 

Shiloh Howells, nine, has lungs so fragile that she is expected to have a risky double lung transplant by the age of 12.

Doctors say the extremity of her condition is far worse than other CF patients her age, having had a lobectomy – surgery to remove part of her damaged right lung, in October 2017.

Her mother, Nichola Howells, 40, said just knowing Orkambi exists is heartbreaking, considering it could preserve what lung function Shiloh has left.

Ms Howells said: ‘It would mean everything to get Orkambi within that time. With a lung transplant, there is no guarantee you’ll survive it. It’s still life-threatening.

‘Her condition isn’t stable and it’s frightening. If she had that drug, within a year who knows what an improvement it wold make.

‘It’s soul destroying for us. It’s money and finance over a life. We are angry and frustrated.’

In March 2018, ex-Health Secretary Jeremy Hunt visited Great Ormond Street Hospital in London and was welcomed by Shiloh who gave him a teddy bear, the hospital’s mascot.

But, according to Ms Howells, as soon as he was told Shiloh was being treated for CF, he swiftly moved on amid the price-war outrage.

Ms Howells said: ‘While she was in mid conversation he stood up and walked away. Maybe he was worried we would bombard him with questions. We were all taken back.’

From the age of six weeks, Shiloh has been having routine hospital admissions every three months to receive treatment to avoid infections.

‘But she wasn’t making the three month mark before getting ill,’ Mrs Howell said. ‘So she now goes to Great Ormond Street Hospital every two months for antibiotic and IV treatment, staying between two weeks and three months.’

During Shiloh’s longest stay, aged eight, she stayed for three months when her lung function dropped to 36 per cent.

Her baseline lung function is 60 per cent, but it used to sit around the 75 per cent mark.

However, always positive and smiling, Shiloh managed to go to school for a solid two months recently – the longest period in two years.

Ms Howells, who has an older daughter called Essence, 20, said: ‘She’s been on the brink but thankfully she picks herself up. But it’s getting harder and harder.

‘Her lungs are already very delicate. To prevent other stories like hers, patients should be given Orkambi as early as possible.

‘CF is all about prevention. We are forever throwing medication at the sufferers to try and make them as comfortable as we can. But Orkambi tackles the genetic side.’

Having just turned two, Lorcán Maguire, of Downpatrick, Northern Ireland, is still unaware of how different his life is to other children’s.

Every day he has 18 tablets, two nebuliser treatment for antibiotics, another nebuliser for a drug to thin the mucus, two fifteen minutes of physiotherapy and vitamins because he can’t absorb them properly from food.

His mother, Jen Maguire, 34, said: ‘It’s a lot for a wee man, and he is still going to die. 

‘The estimate now is mid 40s. But Orkambi has shown to add 20 years to current life expectancy. 

‘It’s such a terrible situation. I wish he had the same life expectancy as his sister.

‘He just thinks his life is like everyone else’s. I hope we get Orkambi before he is aware of the situation.

‘It must be so harder for older people with CF who know the government just don’t care.’

Mrs Maguire, who also has an 11-month-daughter, Saorla, said the condition has ruined Lorcán’s infancy – and it’s only the beginning.

She said: ‘He can play with other children but we are constantly using bacteria gel and keeping eyes opening and ears open. 

‘As soon as you hear a cough or smell a cigarette we would whip him away immediately.’

When Lorcán was diagnosed with CF two weeks after birth, Mrs Maguire and her husband, Dermot Maguire, 45, a social worker, had barely heard of the condition which would change the whole family’s life.

They are on the verge of moving to the Republic of Ireland, where Orkambi has been given to eligible patients since 2017.

Since January, Orkambi has been licenced – but not funded – for two-year-olds in the UK.

Mrs Maguire, who has put her PhD studies in education on hold because she is too stressed to study, said: ‘If nothing has happened by summer holidays we are thinking of renting out our house and moving down there.

‘We are hoping something will happen before then, because it’s not ideal. It’s scary knowing how quickly things worsen with CF.’

Currently Lorcán is trying to fight an infection called pseudomonas aeruginosa, which has been persistently difficult to treat since November 2018.

He’s just returned from a ten day stint in hospital, isolated in a room to avoid contracting more infections from other CF patients.

Drugs were being pumped through an IV line, after three months of antibiotics failed to treat the infection. The doctors are still unsure if it has gone.

Mrs Maguire said: ‘Can you imagine a two-year-old confined to a room? He was screaming and screaming as they put the IV lines in and he shakes every time.

‘It’s worrying because the infection is becoming more resistant to drugs but it causes a lot of damage.

‘Orkambi has been shown to reduce bacteria and hospitalisation by 60 per cent. It deals with the root cause by making the mucus less sticky.

‘It’s not good to pump children with these drugs that only treat symptoms when you could stop something in the first place.’

MailOnline contacted NICE and Vertex for comment, who both said talks are ongoing. 

In a statement, Vertex said: ‘Vertex is in ongoing discussions with NHS England and NICE. We are unable to comment further at this time .’

NICE said: ‘We would welcome re-engagement from Vertex. Vertex, NICE and NHS England met in March and have agreed to continue discussions.’ 

For more information on cystic fibrosis and the fight for Orkambi visit the Cystic Fibrosis Trust. 

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