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.

First Impressions from Atlanta

Today sees the start of the North American Cystic Fibrosis Conference (NACFC), the largest cystic fibrosis gathering in the world, in Atlanta. We've sent four delegates from the Trust to the event and our first blog comes from Rebecca Cosgriff, our new Registry Lead, on her first impressions from being a first time attendee to NACFC
This is my first visit to Atlanta, the home of CNN and Coca Cola. But, more importantly, it's also my first time attending the North American Cystic Fibrosis Conference. After being appointed as Registry Lead back in August, this conference has been an excellent baptism of fire into a 4,000-strong sample of the international cystic fibrosis community.

The atmosphere is positively fizzing over with enthusiasm for helping people with cystic fibrosis to have the same opportunities as those without it. Much of the content over the coming days is rooted in the aspiration for people with CF to be held back no more by a cumbersome medical regime, discomfort, or fear.

As the person within the Cystic Fibrosis Trust responsible for the UK CF Registry, the database that records and reports on the health and care of people with CF, it's been great for me to meet international counterparts. The prelude day to the conference has seen a flurry of Registry-based activity, illustrating the importance of these projects to improve care and outcomes for people with cystic fibrosis.

First up was the Cystic Fibrosis Foundation's Registry Coordinator's luncheon, which saw presentations from the US Registry team on data quality, future plans, and research. It was great to see that that our ambitions for the UK CF Registry are aligned with theirs, with a little friendly rivalry helping to drive impact for people with cystic fibrosis. The dedication of the Registry coordinators was evident; something which is echoed by those responsible for entering and checking data in the UK. Some attendees had been participating in the US registry since its conception in the 1980s!

Later in the afternoon I was able to attend the inaugural CF Registry data harmonisation meeting; an initiative aiming to help international registries work together to evaluate care and detect ways to get the best outcomes. Work of this nature enabled the recent publication of a UK/US data comparison, which found important differences in the outcomes for paediatric patients in the two nations. The meeting saw registries from all over the world, including Brazil, Spain, Australia and of course the UK, represented. This further illustrates the commitment of Registry teams to enable the comparison of international data. We came away from the meeting with new priorities, and plenty of volunteers to form the expert groups that will seek to address them.

With the conference officially commencing tomorrow, I already feel I have the beginnings of a valuable global network, and plenty of great ideas, that can be used to maximise the benefits of the UK Registry when I return to the Trust next week. 

Time To Talk Organ Donation

From December 2015, deemed consent of organs will be granted in Wales in the event that someone passes away, unless they have opted out or said otherwise to their loved ones. As the Welsh government works to prepare people for the change in the law, Kayleigh Old, Public Affairs Officer for Wales at the Cystic Fibrosis Trust, explains why it’s ‘Time to Talk Organ Donation’.

The Cystic Fibrosis Trust is supporting the ‘Time to Talk Organ Donation’ campaign, the Welsh Government’s initiative to encourage people to let their loved ones know their wishes regarding organ donation in the event of their death.

As the people of Wales get closer to the historic move to a soft opt-out system, it is vital that all of us, wherever we live in the UK, talk to our families and friends about what we want to happen after we go.

Losing someone you love can be one of the most emotional, confusing and painful experiences, and being asked about someone’s wishes after they have died can add to that stress and sense of loss. By talking your feelings through clearly with your family, you can remove any doubt they may experience in the unfortunate event of your death, and ensure that your wishes are carried out.

With the Trust’s commitment to improving transplant rates for those with cystic fibrosis, this initiative is very encouraging; we believe that the Welsh Government is leading the way in organ donation. We are hopeful that their new law will bring about awareness and a rise in transplantation rates across the UK, as there will be more organs available for donation.

Currently, one in three people on the transplant list for lungs, dies waiting. Our Hope for More campaign highlighted ways that this situation could be addressed, including a national lung allocation system and expanded use of extended criteria lungs. 

We are continuing to work with organisations that can take this crucial work forward, including transplant teams, cystic fibrosis services, legislative bodies across the UK and the NHS Blood and Transplant Group.

In 2015 the soft opt-out system will become law, and we hope that this will pave the way for the other UK nations. Before then, we must use this opportunity to tell the people we care about most what we believe. As the campaign says, it’s ‘Time to Talk Organ Donation’.

To find out more about the Soft Opt Out, please click here http://www.organdonationwales.org/.