CEO, Cystic Fibrosis Ireland
I began as CEO of Cystic Fibrosis Ireland in 2009. The first priority identified by the Board was to organise a series of meetings around Ireland to hear at first hand the issues facing people with Cystic Fibrosis (CF) and their families.
The fear for adult CF patients was going into hospitals. They often ended up in multi-bed respiratory wards. Sometimes they ended up beside very ill elderly patients who posed a health threat to them (not their fault, of course). In addition, the double-lung transplant programme in Ireland was only managing to transplant about 2-3 patients a year. There had been a major advances in drug therapy during the 1980’s and 1990’s. However, there had been no major medication breakthroughs for CF since the nebulised antibiotic Tobramycin became available in Ireland in 1998.
CFTR drug therapy
It took scientists a further 23 years before the first CFTR drug therapy was approved in Europe in 2012. Most people who took the drug found it transformed their lives because it was the first drug that impacted on the underlying cause of CF. The underlying cause is in fact related to blockages to the inter-cellular transfer of salt and water, which leads to a build of mucus especially in the lungs.
Drug developments and their impact
The positive impact of this therapy was immediate and profound. Word about this quickly spread throughout the CF community in Ireland. Eventually, the news became global about the potential of further research for drugs that could impact on a much higher percentage of the CF population. These began to appear five years later.
In 2017, two further drug therapies were approved by the HSE. The drug therapies had potential to impact on about half of the CF population in Ireland (those with two copies of the F508d gene mutation).
Then, in 2019, came an announcement that had been anticipated since 1989. A drug therapy had the potential to impact on a huge 90% of the CF population worldwide. This drug therapy was approved in October 2019, some five months ahead of schedule in the United States. It could be available in Ireland as early as the summer of 2020.
Resources still needed to ensure progress
The Director of the US National Institutes of Health, Dr Francis Collins, one of the pioneers who led the team that found the CF gene mutation in 1989, stated in a recent editorial in the prestigious New England Journal of Medicine “This should be a cause for major celebration.”
Dr Collins further described the improvement in lung function of patients treated with the triple-combination therapy in the Vertex-funded trial as ‘striking’.
The emergence of these wonderful new medications is to be celebrated. However, they are not a cure and we still need to ensure there is sustained adequate resources also given to lung transplantation. We also need to ensure resources to our CF centres (including Beaumont Hospital’s long promised 20 bed adult CF in patient unit). And lastly, adequate resourcing of clinical staff positions in CF centres.
Multi-disciplinary efforts needed
In short, we are in a period of significant hope for people with CF in Ireland. CF is rapidly moving from a life-limiting to a manageable condition, but not without further challenges to come. Cystic Fibrosis Ireland has been proud to have played ‘a not insignificant role’ in improvements to CF care. Most recently, with the emergence of the National Clinical Programme for CF which came about through our initial advocacy to the Department of Health and the HSE and which is beginning to make an impact.
With all this progress, many are involved. Not least the clinicians and their multi disciplinary teams; the hospitals with CF centres; the HSE and successive government Ministers and the support of the public. If you are interested in fundraising for Cystic Fibrosis Ireland you can find many ideas on www.cfireland.ie. Thank you for previous support.