Living with cystic fibrosis: ‘I am faced with a daily battle to keep well’

(GETTY IMAGES)

Against all the odds I’ve made it to the grand old age of 41 but every day is a grim struggle. For those who don’t know, cystic fibrosis is a genetic condition which clogs my body with sticky mucus, mainly affecting my lungs but also my digestive system and pancreas.

I am faced with a daily battle to keep well, a constant cough and enough medicine each day to fill a whole table. I’m currently recovering from a three-week intravenous (IV) antibiotic treatment for an infection. These happen at least twice a year.

The draining effects of this IV treatment have reminded me how relentless this condition is and how at times it can control my life. But it has given me time to look into this new super drug Kalydeco – the first medicine to treat the root cause of CF.

Kalydeco has been described as ‘transformational’ and ‘game changing’. How exciting that in my lifetime a drug has been produced that actually corrects the genetic fault. It has opened up a new area of genotype specific medicines that are the closest thing to a cure yet for cystic fibrosis. It comes in the form of two blue tablets, which control the symptoms in the lungs and for some of the digestive system too.

The availability of the drug on the NHS in England is currently being reviewed, as is typical, the delay in getting this to clinics is about cost. The delay or ‘block’ was covered in The Independent on Sunday this weekend however it is hoped that the issue will be resolved this week.

Kalydeco only treats patients with CF who have at least one copy of the G551D mutation, there are around four per cent in England, about 350 patients who would benefit from this medicine. Even though I don’t have the G551D mutation, that would benefit from this drug right now, I know of some fellow patients that do and they are frustrated with having this tempting carrot dangled in front of them.

I have the most common type of CF mutation, F508Del, which 90 per cent of people with CF carry, but I want to help because the prescription of Kalydeco would help me in the long run. There are further trials using Kalydeco in combination with a substance called VX-809. The first results from these trials have shown a potentially positive impact on the more common F508del mutation.

After 41 years battling with CF, taking 40 tablets a day and many other treatments, I can’t imagine having only two little pills to take a day. It would also have a massive impact on my life, hopefully prolonging it so that I can enjoy a better quality of life with my wife and son.

I want this drug to be accepted on the NHS, so that the new wave of genetic medicines can benefit even more people with CF. I decided to sign the Cystic Fibrosis Trust’s Kalydeco e-petition because I now know how important this test case is. The petition was handed to Vertex, the drug company that produces Kalydeco on Friday. I hope that both Vertex and the NHS will realise the strength of feeling in the CF community when they meet this week to discuss the cost of providing the drug.

The e-petition is still live, until a final decision is made, so please sign it to support the Cystic Fibrosis Trust’s plea to get this drug into clinics as soon as possible. Together we can all make a difference and breathe life into the lungs of CF sufferers.

For more information about cystic fibrosis visit www.cftrust.org.uk

To read more about Tim Wotton’s experiences of living with cystic fibrosis visit timwotton.wordpress.com

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• Tom Pearson

  Tim — my sentiments entirely. I signed it a few weeks ago having a
  grandson with CF so anything to help the cause. I have been hearing the
  message “we are so close” for 14 year so to have this drug go live is
  exciting, even if it is only for a few people. It is a shame the
  profile of CF is so low compared to Cancer or Heart disease to name but
  two.

  Not sure what mutation my grandson has so must ask this question but hopefully it will lead to greater things.

• Sculptor471

  A young friend has a double G551D inheritance – but has an active life and successful career. It is not clear if Kalydeco trials showed even more spectacular results for this double inheritance – compared to the more common single inheritance G551D.

  Since he was twelve he has dreamed of Gene Replacement Therapy curing CF. Having survived a near-fatal operation – the arrival of Kalydeco offers not only a potentially spectacular improvement in health but would also reduce the future load on NHS facilities.

  It is to be hoped that Vertex and the NHS have found an economic solution to the pricing discussions. The chances of my lottery tickets providing private funding are just too slim.