The drug that gave me my life back

When I first arrived in New York in August 2014 I found myself thinking of the newness of this place while sitting in a coffee shop on Broadway at 6am one morning as daylight rose slowly, sneaking in, casting away the dark of night. I liked to watch the transition, childishly mesmerised by the ritual of it. The miracle of being – let alone being in New York City, let alone being in the moment as the city comes to life – is something that does not get old.

When I moved here to take up a place at Columbia University it was six months since I had started Orkambi, a medicine created by Vertex to treat the cause of the most common mutation of cystic fibrosis. Cystic fibrosis causes a thick, sticky mucus to build up in the lungs, digestive system and liver, and affects other organs also. It eventually results in the need for transplants if the person is well enough to undergo them.

Nothing much changed in my first few months on the drug, and I now think I was originally on a placebo dose. Trials require a lot of patience, trust and guesswork, and the handful of participants in Ireland were not allowed talk to one another about it. In stage three of the trial last year, everyone participating worldwide moved on to some unknown dose of the real drug.

This uncharted magic of new drugs seeping into my system realigned my genetic protein, correcting that which was once thought uncorrectable. My father always said, “You just have to keep going. You just keep doing the work until the cure comes.” This isn’t a cure, but for the first time in history it’s a treatment that works on the cause of cystic fibrosis and not just the symptoms.

Explosion of the now

I found myself sobbing with joy while walking to the university one morning in the February snow. I thought what a great pleasure it was being out in such cold weather, which I would have had to completely avoid before. I realised I no longer lived from month to month, beneath the weight of constant intense medical monitoring and painful evaluation of every single action I took and how that might affect my energy and health for the day. I lived in this explosion of the now.

This is one of the reasons HSE director general Tony O’Brien’s comments last Sunday were so upsetting. He said Orkambi would not be funded in Ireland as it is too expensive. The cost is very high, but it is the HSE’s responsibility to negotiate that price, as they did with Kalydeco – which treats 10 per cent of the Irish population – in 2012. And it is the responsibility of Vertex to work with them. Everyone deserves a chance at the basic freedom I now feel.

One of the greatest gifts Orkambi has given me is the ability to walk places. In New York, walking is vital; taking daily, steep subway steps is a part of everyday life. On mornings before class I walked as an adventurer, scouting out new views and new coffee. I walked, unleashing breath as I did so, and I meditated on the big air that flew back to fill me up. Being able to do this without stopping was overwhelming.

In New York the sweet smell of pretzels wills me to a sidewalk cart. Cute squirrels – all sharp movements and glare in the park – make me pause, and I remember to stop and just breathe. I hadn’t even realised I had stopped thinking about breathing until I started again.

I stayed out of hospital for nine months, which was my longest time in adulthood. I grew occasionally anxious at my new-found freedom, but the wonderful friends I have made helped me. Before Orkambi I chose carefully where my energy went and inevitably let friends down a lot because of fatigue and spontaneous illness. I worked as best I could, but grew frustrated about not being able to keep up with the level of my peers.

Before I started the drug, I had so many lung collapses that my team had discussed a double lung transplant with me, and my window of life was narrowing. My mind was constantly filled with an unconscious timer that counted down the days of infection and measured when I would need to start intravenous antibiotics. I plotted out whether it was likely I would get a bed at that time of year and agonised over the healthcare system. Some days I had to decide between going for coffee with a friend or having a long phone conversation with another.

I couldn’t always do the work I wanted to. I missed birthday parties. I missed dating. I missed life. When life is incessantly interrupted by illness, the last thing you want is drama or having to spend energy explaining why you can’t do something. All of these aspects are very real for people with severe, chronic illness. The world gets smaller.

New York has given me a new life. Although I still have cystic fibrosis and take daily treatments, for someone who was in hospital every five weeks of my adult life, for three weeks at a time, this really is freedom. I miss Ireland often and I miss my parents, but when this happens I remind myself how lucky I am.

In June my parents visited and I got to show them Columbia campus and Radio City Music Hall, but after they left things changed. One thing the drug will not solve for me (not everyone has this problem) is lung collapses, which happen spontaneously because of how progressed my cystic fibrosis is. In June both my lungs collapsed and I was hospitalised.

When lungs collapse there is a high possibility they will again very soon. A few weeks later, at home, one of them collapsed again and the doctor sent me back to my apartment with an oxygen tank and instruction for bed rest. A week later the pain got much worse and I found myself immobile in an American hospital with a chest drain and various other tubes sticking out of me. During this I was lucky the American Ireland Fund was so supportive and made me feel safe so far from home in a life-threatening situation.

My mum flew over to be with me and her presence was medicinal. The situation was grim but things improved. I considered whether I might have to come home, but I knew I wanted to finish my master’s at Columbia even though it might take me longer than my peers. I had come this far, and the prospect of getting back to campus became the prize.

I needed a procedure whereby a chemical was injected into the lung to stick it back to the lung wall, which will hopefully stop it from collapsing. It’s usually done just once, but there was a need to do the procedure three days in a row. This shattered my body, but I listened to some chanting Buddhist monks to stay at peace.

An incredibly kind Irish physiotherapy graduate offered her support for free, came to my home and helped me mobilise and move the mucus that was stuck in my lungs, so that when school started again in 10 days I would be able to walk the handful of blocks to the classroom.

My body felt worse than it ever had before starting the drug or any time I could remember. I had to cut my course load in half and was sleeping with oxygen on overnight to top up my reserves for the day. In July the US Food and Drugs Administration approved Orkambi, and I was well enough to receive my first commercial dose at the start of November.

Miracle drug

This was a miracle for me. The higher dose took effect immediately and my body purged itself of the mucus created by cystic fibrosis. I had hot flashes and intense insomnia. After one week I could breathe more deeply than I can recall in adulthood.

I stopped having to use the oxygen to top up my reserves. The mucus is now consistently much thinner and there is less of it. My chronic cystic fibrosis-related sinus problems – which often instigated infection in my lungs – are gone.

The price of Orkambi needs to be driven down so it is accessible to the people it is designed to help. The drug has been proven to increase lung function and decrease the need for hospitalisation by 40 per cent.

I know two other Irish people on the trial who have been out of hospital since starting. That freedom is different for everyone. It might be the difference between being able to go shopping for groceries or not; or the difference between being able to go to college or not.

What’s scientifically proven is that the drug allows people with cystic fibrosis to reconnect with life again in a significantly improved way. The earlier people get the drug, the less lung damage done, the fewer hospitalisations are needed. Ultimately fewer double lung transplants will need to be performed. In the long run it saves money but, most importantly, it saves lives.