SPOTLIGHT: Trikafta: A Game Changer
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s we anxiously await the approval of Trikafta here in Ireland, we are delighted to share with you one member's experience on this groudbreaking new therapy. This person with CF, who has chosen to remain anonymous, gives us a sneak preview of the encouraging results they have seen over the past six months. A member of CF Ireland, this PWCF is currently living in America. Further information on Trikafta is available on page 4. It is too early to be conclusive about the long-term impact of Trikafta but the future appears bright.
At the heart of this piece, I want to show you what Trikafta has meant for me. Because I want anyone reading this who will be eligible once it is approved, to feel hope. Trikafta is a game changer. On the morning of the 3rd of December I sat at my work desk and took my first dose of Trikafta. I was on the phone to my mom talking about how nervous I was to take it. I had made myself a breakfast with the requisite level of fat – a bagel and cream cheese if you’re asking – and was just peering down at these two, small, light orange tablets. I had read so much about this drug. My work is in science and I have been avidly following Vertex’s developments, clinical trials, announcements and even stocks for years. I never bought stocks though, shame on me. My friend, however, on my advice, did buy some, and he is now forever indebted to me. I watched over the past five years but my gene combination was such that neither Kalydeko nor Orkambi/ Symdeko/ Symkevi were for me. So I was one of the ones who had to wait, but here I was, my day had come. I popped those pills and laughed with my mom at my dramatics. To give some background, I’ll tell you the top notes about me and my journey. I was born in Dublin thirty five years ago. At six weeks old I was diagnosed due to the fact I was growing in length but losing weight and crying around the clock. My poor parents! Once I got started on pancreatic enzymes it was pretty much plain sailing. All the way through school I had no infections and was blessed to never need IV treatments. I was extremely active through ballet and tap dancing, playing lots of sport – mainly hockey and basketball, and having a mom who was aware of cross infection before that was even a thing. I had a great start in life and am forever grateful for that. I went to UCD for third level and it’s safe to say I probably enjoyed the social side of college life a little too much! It was around that time I started to need IV antibiotics. At first this was once a year but when that frequency started increasing I realized that if I didn’t take action, it was going to get really serious.
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Spectrum / Summer 2020
