Cystic fibrosis patients say new drug has saved their lives
Three cystic fibrosis patients at the specialist Liverpool Heart and Chest Hospital have described how they have been saved from serious illnesses and potential lung transplants, thanks to a new drug therapy, Kaftrio.
In total 222 patients at LHCH have been treated with Kaftrio, a triple drug treatment, transforming their lives with all reporting a significant improvement in their health.
It comes after NHS England announced on 30 June 2020 that a deal had been agreed for Kaftrio.
The Trust’s Clinical Lead for Respiratory Medicine Dr Martin Ledson said: “I did not think it would happen in my lifetime; all the patients suitable for the treatment have had huge benefits.
“If it is possible, we are as pleased as the patients – we have been watching and waiting for these therapies to be developed and made available and now it is a reality, it is a wonderful success. Also, due to flexibility in the criteria of eligibility for treatment agreed by NHS England as part of the deal with Vertex Pharmaceuticals, we are now able to offer it to more of our CF patients.”
Cystic fibrosis patient Sarah Kay of Crewe missed much of her secondary school life due to repeated stays in hospital with serious chest infections and pneumonia. At the age of 15 she was in hospital for two weeks out of every month in the year.
The first time she was admitted to hospital with a collapsed lung she thought she was on a downward, final spiral. She was 28 and had lost a friend with cystic fibrosis the previous year.
“I went into hospital thinking this is it for me – I always thought I would be dead by the age of 30,” she said.
It was the first of six lung punctures in quick succession. “Having repeated chest infections caused by cystic fibrosis leaves scar tissue in the lungs and when that membrane rubs against the ribs it can wear away and cause holes. Each time, I had to have chest drains which are extremely painful; it involves pushing a tube through your ribs to allow air to escape – and you have to be awake.”
To prevent further lung punctures, Sarah endured an even more painful treatment, in which her body was repeatedly tipped upside down and an adhesive placed onto one of the lungs to bind it to her ribs, to stop holes forming.
When the other lung needed similar treatment Sarah persuaded her surgeon to carry out a procedure that was deemed high risk, removing layers of pleura and ensuring the second lung was also fixed to the ribs.
“There was only a 50-50 chance of surviving the op but I was so happy to be getting it, I couldn’t have gone through the other procedure again.”
Six months later Sarah suffered her worst ever chest infection which brought her lung capacity down to just 27 per cent. She became completely dependent on oxygen and on other people to help her.
By now she had given up her job as an appointment clerk in Leighton Hospital’s MRI scanning department, as she was too weak and exhausted.
“After recovering from that chest infection I was knackered all the time; I would wake up gasping for a nebuliser. Even when I was stable I wasn’t well enough to work as I could get ill very quickly and my lung capacity stuck at 39 per cent.
“Psychologically I was massively affected by giving up work, I didn’t feel part of society, I could only stay at home and look after myself; I felt out of the loop, I had nothing to talk about!”
Sarah and her husband’s first attempt at IVF failed and Sarah had started another course of treatment to try again, when her consultant Professor Martin Walshaw at Liverpool Heart and Chest Hospital offered her the drug Kaftrio.
“I hadn’t heard of it but I told a friend with CF who was super excited and told me to do some research. I couldn’t believe what I read on chat forums about people having amazing recoveries and I didn’t want to get my hopes up.
“I agreed to take it even though it meant putting off the IVF and I was one of the first in our clinic to be given Kaftrio, in November 2019.
“I felt the impact in just six hours. I started coughing up loads of mucus; usually it is so sticky and thick it doesn’t come out but this was like water and kept coming. I remember this strange sensation and I realised I was feeling air at the bottom of my lungs, something I’d never felt before.
“It was incredible, very exciting but also overwhelming to get my head round it, after always thinking I would die before my mum and accepting it.
“It has changed my view of the future and it’s very freeing; the pressure is off for having a baby, it is no longer so time sensitive and I can get pregnant when we are ready, whereas before it was up to the CF.”
Sarah and Stephen are now going to try for a baby naturally, having seen lots of women on Kaftrio becoming pregnant. “It seems to be the untold story of Kaftrio, so many pregnant women are appearing at clinic which has never happened before!”
Sarah also suffers from CF-related diabetes but has been able to reduce her insulin intake by two-thirds.
“Kaftrio is the most amazing gift that the NHS and scientists could have given and it’s not just to one person it’s to 90 per cent of the CF population.”
Her husband Stephen Kay has embarked on a 125-day ‘Furthest Points Challenge’, cyclng 16-20 miles each day on a static bike at home to raise money for the LHCH Charity. This mirrors his motorbike tour in 2010 which visited the four furthest compass points in the UK mainland, a total of 2,500 miles.
Keen cricketer James Wojda of Whitchurch, Shropshire, was in his mid-teens when the life-long condition started to affect his health and his performance on the cricket pitch. He battled through with a positive mindset and determination not to let cystic fibrosis rule his life. He became a professional cricket coach and continued to play the sport, but his condition deteriorated and four years ago aged 25 he suffered a slow puncture to his lung.
Two years later he suffered another punctured lung, this time as a sudden emergency which left him unable to breathe. Fortunately he had oxygen at home which kept him alive before being rushed to hospital.
“I had to stop playing cricket, move back home with my mum and dad and get my head round the fact that I wouldn’t be able to run my own cricket coaching company,” said James.
Over the coming year he spent a total of 22 weeks in hospital, after 11 admissions. In March 2020, he was in hospital and at his lowest ebb.
“It was a scary period. I was in hospital with the punctured lung and my veins had also collapsed so a port had to be put into my left arm, which wasn’t straightforward. I was told that in another six months I could be going on to the lung transplant list.”
Then came a moment when he knew everything was about to change for the better.
“I knew about the drug Kaftrio and the team at Liverpool Heart and Chest Hospital had told me they had applied to the company Vertex for a license to give it to me in exchange for my data. One morning they came in for the usual ward round and my mum was with me, as she visited every day.
“The consultant Professor Walshaw was with the pharmacist and two cystic fibrosis specialist nurses and he held up a white paper bag and said he had something for me – a bag of Kaftrio tablets.
“My mum was crying, the nurses and pharmacist were emotional and I was emotional – I knew that was a changing point in my life.”
The effect was almost instant. Two days later he suffered a more severe than usual coughing fit and vomiting. The next morning when he woke up he didn’t need to cough – for the first time in his adult life.
“Every morning I used to start the day coughing up mucus which had settled on my chest overnight and then being sick. Now, I don’t cough at all – in fact I haven’t coughed since three days after starting on the tablets.
“My spirometer reading of lung function at its lowest was 22 per cent and the last time I measured it, it was 49 per cent. At one stage I didn’t think I would last another year; now, I feel like me again.”
James is once again playing cricket and working as a professional cricket coach; he has been taken on by Shrewsbury Cricket Club to coach in the prestigious Birmingham Premier League.
“Kaftrio has completely changed my life. Before I was just surviving, now I am living my life to the full; my life has been given back to me and I can’t thank the hospital team enough for what they have done, researching and pushing for me to get the drug.”
James vlogs about his experiences on his YouTube channel, James and Cystic Fibrosis.
Listen again to James speaking on BBC Radio Shropshire breakfast with Adam Green - skip forward to 2:35:10.
At the start of 2020 Jody Lewis, 31, from Pen-y-lan near Wrexham, but treated in Liverpool, was fighting for her life having contracted flu. Her lungs were weak and damaged after two years of multiple hospital admissions to treat blood clots and narrowed veins.
She faced the prospect of a double lung transplant but surgeons were reluctant to put her on the list while she was so frequently ill.
Jodie said: “At the time I had spent two years of my life in constant agony from the pressure build up in my head due to the narrowed veins. My lungs were rapidly failing; I was on continuous intravenous drips at home, I was having constant chest infections and was unable to properly clear my lungs. I had to use oxygen to walk just a few steps.
“Lung transplant was now becoming a very serious and very scary option. I was 29 years old and facing a very bleak future, I had even begun planning my funeral in secret as there was no guarantee I would survive the transplant, yet alone make it past one year.”
Jody was a keen horse rider and animal lover and owned three horses and five German Shepherd dogs. She had been sporty and fit and competed regularly in show jumping.
She believes that one of her pets played a part in her recovery. She had been told by her physio that she couldn’t leave hospital because she needed such a high level of oxygen. Then one night she got a call that her oldest dog Daisy Duke was very sick.
“I had to be with her so my dad picked me up and I rang the vet and asked her to meet me at home. I was hooked up to oxygen tanks in the car home and I remember having a sick and empty feeling in my stomach.
“As soon as I saw Daisy Duke lying by the fire I knew she had to go and she was put to sleep that night. I lay on the floor cuddling her and crying, I felt so guilty I’d not been there for her in her final months.
“We buried her in a favourite spot; I didn’t return to hospital as I didn’t want to spend another day away from my other dogs.”
Three days later Jody had a call from hospital to tell her here was a supply of Kaftrio waiting for her.
“Within a week of taking the tablets I no longer needed oxygen at rest; my sats had shot up to 94 per cent, a number I hadn’t seen for a long time. I was putting on weight and my energy levels were rising. I was waking up in the morning actually feeling refreshed and not drained.
“In December 2020 I recorded my highest lung function test for five years at 47 per cent; at my lowest it had dropped to just 24 per cent, in January 2020.
“I lost nearly three years of my life battling one complication after another, facing so many setbacks and seeing myself wither away and change from being energetic, bubbly and happy to a very lost, angry and frail person.”
Jody now sees the possibility of having a family – which she had previously ruled out.
“It took a long time for me to get my head round the fact that I wouldn’t be able to have kids and now, thanks to Kaftrio, it is a possibility. It has saved my life and given me a new lease of life, it is like a miracle.
“It is so important to keep a positive mindset and never stop fighting; I am proud of myself for not giving up even when all the odds were against me. I’d like to tell anyone else who feels like they are sinking to dig deep and just keep on fighting, whatever happens.”
Jody is planning to use her experiences combined with her love of animals, to help others. She has joined the volunteers at Therapy Dogs Nationwide and is undertaking training in counselling for children and young people. She is looking forward to working with her dog Echo to help children with illnesses and learning disabilities.
“I cannot thank my wonderful family and friends enough for all their help and support; and the amazing cystic fibrosis team at Liverpool Heart and Chest Hospital.
“I believe I had to have this experience to get to where I am today, in a position to help other people. I am now ready to begin the next chapter of my life with the people who were always meant to be there.”