Cystic Fibrosis Gene Therapy Trial testament to the cf community as a positive, patient and persistent partner in research

I hope you will have seen or heard the news today of the positive results of a gene therapy trial for people with cystic fibrosis. And this is how The Guardian is reporting it.

Although it is early days and the improvement shown in patients only a modest one, it is nonetheless a very hopeful sign of how this approach may help people in the future.

I have no direct experience of the disease, but one of the most affecting and poignant patient stories I have witnessed was by a young woman with cf at an event at the political party conferences many years ago.  It has stayed with me ever since.  It has caused me to have a particular emotional interest – if I may call it that – in the condition.

The trial was conducted by the Cystic Fibrosis Gene Therapy Consortium which brings together scientists in the Universities of Oxford and Edinburgh with Imperial College, London.  They were formed in 2001 at the initiative and with the support of the Cystic Fibrosis Trust (CF Trust). The Trust has invested £40 million in the consortium since then and continues to do so (it announced a further £500,ooo funding just earlier this year).  But the funding environment for these sorts of projects is never easy and a few years ago the Consortium formed ‘Just Gene Therapy’ to bolster existing funding from the National Institute for Health Research (NIHR), Medical Research Council (MRC) and others.

So, the back-story of this important news today is one about the length of time it takes to do this sort of work, the importance of collaboration and the pioneering role that medical research charities have in supporting ground-breaking science.

But, above all, it is a testament to the cf community’s as a whole.  Their positive, persistent and patient backing and support through their donations.  The patients who took part in the trial, some as young as 12.  Without them, we wouldn’t be hearing this news today.

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