Saturday 20 December 2014

'I believe things can only get better for people with CF'

Orla Tinsley was one of only 1,100 cystic fibrosis sufferers in the world to try out a new medication for the debilitating genetic disease. It has changed her life and she believes the future, finally, is bright for CF patients

Published 14/07/2014 | 02:30

Orla Tinsley. Photo: Ronan Lang
Orla Tinsley. Photo: Ronan Lang
Orla Tinsley: ‘This drug is not a cure for CF, but it is one hell of a start. It is slowly and surely changing my life.’ RONAN LANG

Drug trials are tricky things. You have to work hard to get on them, you have to work hard to stay on them and you can tell next to no one about them. It's now almost a year since I began treatment in what I believe is one of the most important trials in medical history. The results, which were published earlier this month, have proven that 22 years of hard work, investment and research has paid off.

The combination of the drugs Lumacaftor and Ivacaftor for people with cystic fibrosis who have the double delta F508 gene, was found to decrease infective exacerbations by 30-40pc. This means the 1,100 of us across Europe, North America and Australia who were lucky enough to be on the drug trial have required dramatically less hospitalizations in the past 11 months.

After living with a degenerative illness for 27 years, this type of change is life-altering. There is now more time for life, for adventure, for love and for all those things any person could want.

Cystic Fibrosis is a genetically inherited disease that affects everyone differently. It mostly affects the lungs and digestive system but as it progresses it becomes much more complex. While it is possible to have a normal life in many aspects, because the degree of illness is different for everyone, the end result is usually the need for a life-saving double lung transplant which many people die waiting for.

The results of the Vertex Pharmaceuticals trial is an exhilarating injection of real hope for all those dealing with CF across the world. Personally, the scientific results matched up with how I had been feeling for the past 11 months.

There were two divisions of the trial, which were each then divided in three. Patients participating took one drug - Ivacaftor - and either a low or high dose of another drug - Lumacaftor. Patients in the third group received a placebo. After 24 weeks, lung function had improved in all four groups taking the combination drugs. Lung function fell a little in the placebo group. The study found the drugs reduced infections from 30-40pc meaning less time in hospital and a better quality of life

Until now all medications for CF were essentially treated the symptoms of the illness. The combination of Lumacaftor and Ivacaftor changes the outcome at a cellular level and by lessening infections and the need to be hospitalised it changes the progression of the illness.

I started the drug trial in August 2013. Participants were chosen by strict medical criteria and signed legally binding contracts. Only close family and friends could know, any deviation from this could jeopardise everyone's place and the study's outcome. It was an exhausting experience but one I was determined to see through.

After my first dose I recorded everything I felt physically. I was waiting for the big change, but it didn't come. Still I began zipping around, as though enamoured with new life, which I now understand was most likely what is called 'the placebo affect'. I wanted so badly to believe I was on the real deal that I imagined I was. The entire experience was like living in a bubble of precaution and perfect planning because of the drug taking time schedule. There was no room for error.

A month later, while leaning over to pick something up off my bedroom floor, a searing pain shot through my chest. A trip to the hospital confirmed a lung collapse, as can happen in CF.

This meant total lock down and hospitalisation. It also meant one of two things was happening, either the revolutionary drug would not work for me, to the extent that it would make an actual difference, which was a devastating thought. Or, I was on the placebo. I still don't know for sure.

What I do know is this. The determination to stay positive while facing the reality that the miracle may not be for me was hard work. But, of course, attitude is half the battle. My friends and family and medical team during this time really helped me stay positive. The only option was to take life into my hands and plan for the future. I applied for a Creative Writing course at Columbia University in New York that January. I wrote my application while being treated for severe flu with a consistently sky high temperature.

The thought of New York was my lifeline. The many cups of tea and great encouragement from the ward nurses and my medical team certainly helped too. I got the application in just before the deadline with twenty-nine minutes to spare. Five days later, the temperatures stopped. Soon after, the new stage of the trial started. Now everyone, including me, would be on some unknown dosage of the actual drug.

It was in March as my birthday came around that I realized I was still out of hospital. Not only was I out of hospital, I felt well. That same week I got a call from Columbia University offering me a partial scholarship to study on the course of my dreams.

My mind was blown at the new possibilities. I kept waiting for the other shoe to drop. But by May I was still well. In June, I travelled across Europe in constant 20-degree heat to speak at the European Cystic Fibrosis Conference. For the first time in adulthood the heat did not affect me. Instead of becoming breathless after 48 hours in heat, I felt normal.

On a train in the middle of Germany I found myself crying with happiness. This was a real miracle of life, one that would not be reversed. I could take this new life with me on my adventure to Columbia and wherever I went after that. Things that were so uncertain before seemed within reach. And there was something else too, something immeasurable by science, best described as a consistent sturdiness in the body, a strength that wasn't there before that is there now. Maybe too it is that great hope that so often as a child my Dad would tell me about. He would remind me a cure was coming and that I had to stay well and fight hard to be ready for it. This drug is not a cure for CF but it is one hell of a start.

The new drug is slowly and surely changing my life. I don't know for how long it will last, whether it will continue to work or perhaps I will plateau and remain at a certain level of wellness. Whatever happens I am grateful and thankful for this miracle every day. The cycle of infection that has caused my late teenage years and adulthood to need hospitalisation every six to eight months has been broken.

Both professionally and socially I can commit to things. I stopped using a calender a few years ago because I always felt like I was missing out. A few months ago I went out and bought one and now it's hanging in my kitchen.

For just under 80,000 people with Cystic Fibrosis worldwide this new drug means new possibilities. Studies for second-generation correctors are getting under way.

Things can only get better for people with CF worldwide. For people with Cystic Fibrosis in Ireland who have watched the awareness levels and incredible support and understanding of Ireland's most common genetically inherited illness rise over the past ten years, this drug is nothing short of a miracle.

It should take eight months to be approved by the FDA and EMA. Cost negotiations will start which will be watched closely by CF advocated worldwide. Here in this time and space all things are possible for a brighter and better future.

*If you would like to support Orla's journey to Columbia University please visit here: www.friendsoforlat.com

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