SPOTLIGHT: Kaftrio Life Before & After!
T
omas Thompson is a PWCF and former patient advocate for Cystic Fibrosis Ireland. A much respected member of the CF Community, Tomas is now working to research and develop medical devices to improve CF care. In this article Tomas talks about the timeline and development of Kaftrio and gives his own personal perspective about the impact of this new and innovative drug therapy. This article outlines one person's experience. As with all drug therapies, each person will have a different impact from Kaftrio. Further information on the roll out of Kaftrio can be found on page 2 or online at www.cfireland.ie
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am a child of the late 70s and was born when the outlook for PWCF was very different than today. Back then CF was mostly seen as a childhood condition as many PWCF didn’t make it to adulthood, those that did had a very different quality of life to PWCFs today. The treatments available when I was growing up in the 70s & 80s were limited and the research into the CF was only at its infancy. Members of CF Ireland at the time just like today, did all they could to push to speed up the development of new facilities and treatment. In years gone by and prior to understanding of the risks of cross infection, all CF Associations worldwide brought children with CF together for summer camps and similar joint activities. CFI was no different and one initiative was that every year CFI would rent a plane, fill it with as many CF families as they could, flying them to Lourdes to take ice baths and pray to St. Bernadette as this was seen as the best option of a cure at the time! To many this might seem strange today for many reasons, but these families formed the backbone for many years of organisations such as CFI, CF Trust & CF Foundation (CFF) in the US, who raised the funds by any means necessary which without we would not now have the treatments we have today. In the 90s the research sped up around the world, but for PWCF and their families it was slow, with many highs and lows over that decade as promising research into gene therapy failed to bring the desired results and the dreams of the late 80s when they discovery the CF gene was fading, as one approach after another was failing to translate in to an effective treatment. A vast effort was spent on research in the 90s to little effect, but in the early 2000s the US Foundation took a new approach headed up by Robert Beall, he went searching for a drug company that would try to take on CF head-on. That search led him to Aurora Biosciences, a San Diego company. In 2000, the CFF agreed to fund drug discovery work in cystic fibrosis at Aurora with $47 million investment over five years, this is the single most important investment made in CF since the discovery of the CF gene, as one year later, Vertex bought Aurora. www.cfireland.ie
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