From Atlanta to the Summit

Last week saw the annual North American Cystic Fibrosis Conference take place in Atlanta, USA. The world's largest gathering on cystic fibrosis saw clinicians, researchers and carers came from all over to discuss the latest developments and progress towards beating cystic fibrosis for good. The Trust sent over four delegates, and Ed Owen, the Trust's Chief Executive, offers his thoughts on the outcomes from the three-day event.

“I have no doubt that we will reach the summit.” These were the words of Dr Mike Boyle, associate professor of medicine and director of the Adult Cystic Fibrosis Program at the Johns Hopkins School of Medicine in Baltimore, speaking at the 2014 North American Cystic Fibrosis Conference last week comparing the extraordinary progress that has been made in beating cystic fibrosis with the challenge of climbing Mount Everest.

For Mike, the discovery of the cystic fibrosis gene in 1989 by researchers from the US and Canada – with a little help from our very own scientists here in the UK – was the equivalent of reaching Base Camp. Since then, we have been moving up the mountain with significant discoveries.

The development of Kalydeco as the first therapy to treat the basic genetic defect, and the pipeline of new combination therapies are major breakthroughs – and other work like gene therapy, stem cell research and genetic editing will get us closer to reaching our goal.

Tragically, these developments cannot come soon enough for those living with the condition – and, for many, they will have come too late.

But these advancements are testament to the extraordinary power of the cystic fibrosis community.  When people with cystic fibrosis, their families, clinicians, scientists and others work together, we can and do change the world.

And that community is truly global, as witnessed at last week’s conference. More than 4,000 people from across the world converged on Atlanta to discuss the latest developments in research and care, and to confirm our collective focus on beating this cruel condition.

The cystic fibrosis community here in the UK and the Cystic Fibrosis Trust play a key role, not just because we represent more than a 10th of the global CF population; but because of our active and informed community, our dedicated clinical professionals, our expert scientists and established industry base.

The combined resources of the UK, Europe, North America and Australasia are massive and we therefore took the opportunity to meet key people in the CF world to build new collaborations. This included catching up with a number of sister organisations, including the Cystic Fibrosis Foundation (CFF).

Bob Beall and Preston Campbell have led the CFF for well over two decades, and have done more than many to advance our collective cause.  We discussed a series of potential exciting collaborations in research and care which I hope we can bring to fruition. They were impressed by our investments in research on pseudomonas, NTM and other projects and want to work closely with us.

We also met many of the established pharmaceutical companies including Vertex, Gilead, Forest, Insmed and Roche, as well as smaller companies like Verona with which we are hoping to build closer links to develop new drugs and therapies. Also centre stage in our discussions with industry and clinicians were our plans to develop a new model of care with remote digital technology enabling people with cystic fibrosis to take greater control of their lives and improve their health and wellbeing.

There was disappointment in Atlanta that the UK Gene Therapy Consortium was unable to present the results of its Phase II study. This is work we in the UK cystic fibrosis community have given so generously towards over many years and there is great anticipation of the outcome. But there was understanding across the conference that it was vital that the data, when published, should be robust and complete.

In gene therapy, as with so many areas, collaboration is essential to make progress. That was the constant theme of the Atlanta conference and, with the support of the UK community, the Cystic Fibrosis Trust will play a leading role in improving and transforming the lives of all people with cystic fibrosis.

I return to the UK more determined and more confident than ever that we can reach the summit and achieve our ultimate goal of beating cystic fibrosis for good.  It won’t be tomorrow or even in the next few years. But we will continue to make great progress and, with the right support, we will get there.

For those who want to see more from NACFC, you can now watch recorded videos of the live streamed sessions.