Praising Arizona - Looking back at NACFC 2015

The past four days saw Ed Owen, Trust Chief Executive, lead our delegation to the North American CF Conference in Phoenix, Arizona. Here he gives us the highlights from the largest global gathering in the fight against cystic fibrosis.

As the 4,000 clinicians, health professionals, scientists, company reps, families and charities like ourselves pack up and head homeward across the globe, it’s worth reflecting what the 29th annual North American CF Conference has told us about the state of play in our international effort to beat cystic fibrosis.

Rebecca Cosgriff, Registry Lead, presenting at NACFC

The collective determination and purpose remains very strong and was evident throughout the four days of meetings, workshops, speeches and discussions. If anything, the sheer excitement and wonder generated by the scientific breakthroughs of just a few years ago has given way to a cool confidence at what can be achieved across a range of fronts.

This is matched, of course, with a realistic view that there is a long way still to travel before our job is done. But distant hope has genuinely shifted to steely belief and this conference showed how we, as an international cystic fibrosis community, are transforming the character and face of this condition.

A good illustration of this is to witness the attendance of the multitude of pharmaceutical and biotech companies here in Phoenix. Barely a decade ago, much of industry would have avoided investing in a ‘rare disease’ like cystic fibrosis. Today, you can’t move for industry talking about their development of new therapies, devices and diagnostic tools targeted at those with the condition.

All this is good news but not without its challenges. For example, Vertex announced this week further news on its pipeline of precision medicines with planned trials of new compounds to develop ‘triple therapies’ aimed at correcting the genetic defect of those with one or two copies of the F508delta mutation.

But, of course, we in the UK are soon to confront the more immediate challenge of ensuring Orkambi – a mere double therapy of ivacaftor and lumacaftor – is made available to those who can benefit.

A key figure in the development of the pipeline of gene-modifying therapies now coming through has been the Cystic Fibrosis Foundation (CFF)’s long-time President, Bob Beall. After 35 years in his post, he has made way for his deputy, Preston Campbell, and this conference was the opportunity for the cystic fibrosis community across the US and worldwide to mark his extraordinary legacy.

It was great to see him, and I was delighted to present himwith a bottle of fine Scottish whisky to demonstrate the thanks of the UK CF communityfor his work. With the Foundation’s therapeutic development programme, Bob has made a massive contribution to the changes we are seeing today.

But, as I said, our work is very far from done, and while we were here we sat down with Preston and his senior team to discuss a range of collaborations with the Foundation, including the prospect of longer-term innovation to develop a fundamental cure for cystic fibrosis using stem cell and gene editing techniques.

And alongside this transformational activity there is the needs of people with cystic fibrosis community today, and it was refreshing to hear Preston commit the CFF to a programme of activities to better engage those with the condition, to hear the diversity of views and experience of the people we are here for.

As I said in my speech to the UK CF conference in Manchester last month, cystic fibrosis for those living with it is not primarily a clinical or scientific issue, it’s a life issue – and we need to understand that in everything we do to help remove the barriers that prevent people with CF living the life they want.

Fighting for a life unlimited by cystic fibrosis, today and tomorrow, is what we are about, and I am pleased to report from the desert landscape of Arizona that this fight is alive, strong and growing.