CF Unite - one participant's account

As Dr Matthew Hurley discussed in his blog post last week, the first Cystic Fibrosis Unite event took place on Saturday. This was a virtual conference hosted by the University of Nottingham and funded by a Wellcome Trust People Award, discussing Kalydeco. Rose Donnelly, Executive Assistant to Chief Executive Ed Owen, tells us all about it.

The conference was moderated by Professor Alan Smyth, who leads the paediatric specialist cystic fibrosis service in Nottingham, and David Noonan, who has cystic fibrosis. The speakers were Dr David Sheppard from the University of Bristol, Professor Stuart Elborn from Queens University in Belfast, Dr Michael Boyle from Johns Hopkins in Baltimore and Ed Owen, CEO of the CF Trust.

Dr Sheppard began by giving an overview on CFTR (cystic fibrosis transmembrane conductance regulator), explaining what it does, why it doesn't work in people with CF, and what ivacaftor (Kalydeco) does to it. I particularly liked his analogy that ivacaftor affects CFTR like oil on a rusty turnstile - it makes it easier to open and helps it to stay open for longer.

Stuart explained that previous research had shown that milder symptoms of CF are associated with mutations where there is some CFTR function leading to a stratified medicine approach, where specific therapies are designed for specific patient cohorts. The Phase III ivacaftor studies showed very encouraging results in terms of lung function, sweat chloride, weight gain and frequency of pulmonary exacerbations.

Mike's presentation focused on F508del, the most common mutation in people with CF, and the research being carried out by 23 centres worldwide combining ivacaftor with VX-809. Approximately 87% of people with CF worldwide have this mutation, and a therapy successfully treating it could have an enormous impact. Vertex plans to start clinical trials in 2013.

Ed feels the success of ivacaftor represents a paradigm shift in terms of discussion and discourse in scientific approach. Forbes has described ivacaftor as the "most important drug of 2012" and the rapid approval and positive funding position will create more investment. The strategic decision made by the Cystic Fibrosis Foundation was instrumental in developing the drug and the role the patient voice has played cannot be underestimated.

Finally, David Noonan gave a personal account of Kalydeco. After 64 days David has gained 20% lung function, and now feels energetic and has put on weight.

Throughout the session questions were submitted by the virtual audience.

If you were unable to join the event online, you can see a recording on the CF Unite website: www.cfunite.org.