Friday, 14 November 2014

A cystic fibrosis 'cure'? - Understanding this week's research news

Following recent news reports of a ‘cure’ for cystic fibrosis being within reach, Cystic Fibrosis Trust Chief Executive Ed Owen discusses the promising research at the centre of the story, and the need to think big but stay grounded.

Nothing is more guaranteed to get all of us excited than stories of a possible ‘breakthrough’ or ‘cure’ for cystic fibrosis.

So a news item on Channel Four News this week heralding a research project led by Dr Anil Mehta in Dundee as a possible cure for cystic fibrosis has understandably caused a wave of interest among many of us affected by the condition here in the UK.

In a blog on Tuesday accompanying the news piece, the veteran broadcaster Jon Snow described the research as a “Eureka moment” that offered the hope of a “cystic fibrosis cure”. Many people called our office to find out more, dozens tweeted and hundreds posted messages on Facebook at what appeared to be a remarkable moment.

So amid all the excitement, what’s the reality?

First, it is important to note that no research paper has yet been published so we at the Trust have not been able to see the detailed analysis. But the facts according to the University of Dundee’s press statement are that in an early stage clinical study undertaken in Italy, medics trialled the combined use of two drugs already licensed for other conditions - Cysteamine, and epigallocatechin gallate (EGCG) – involving 10 people with cystic fibrosis with two copies of the DF508 gene.

The University statement says that the study found that the two drugs given together reduced inflammation in 9 out of 10 of the patients’ airways and also dramatically reduced their sweat chloride levels. Obviously, then, the research looks promising. But it’s at an early stage trial and used only a very small sample.

I had the pleasure of speaking with Dr Mehta on Wednesday and he agreed with me that talk of a potential cure was misplaced. He acknowledges that his work is at an early stage but wants it to now go forward to a larger Phase 2 research study involving 120 patients. He is planning to submit an application to the Government’s Medical Research Council (MRC) which supports this kind of clinical research in the UK, although there are a number of regulatory hurdles the work needs to overcome first.

As a result of the extraordinary support we receive from thousands in our community, the Cystic Fibrosis Trust is currently investing in a wide range of research projects aimed at improving and transforming the lives of people with cystic fibrosis. This has helped fund earlier stages of Dr Mehta’s work and our new Venture and Innovation Awards created last year are targeting funds at projects like this one as a way of bringing in even larger sums of money from the Government and other quarters.

Dr Mehta and I discussed these funding arrangements and – once we have analysed the full details of this work – we stand ready to offer such an award as part of the process of bidding for MRC funds.

But, three days on from the story being broadcast, I confess to feeling disappointed at the way it was covered.

Talk of breakthroughs and cures make great headlines. But such hyperbolic coverage risks the unwitting manipulation of the emotions and passions of those of us desperate to believe that there is an imminent prospect of being freed from the terrible clutches of cystic fibrosis. Put bluntly, such stories bring into sharp focus the stark contrast between longer life and an early death.

I say this not to pour cold water on all our hopes because this particular project looks promising – and I am hugely optimistic about the wider future for cystic fibrosis. Kalydeco is already correcting the basic genetic defect of cystic fibrosis for four per cent of people with the condition in the UK – and, with the active support of the cystic fibrosis community, I believe we can help ensure that new transformative therapies and treatments will be introduced over the next decade that move us closer to our goal of beating cystic fibrosis for good for all.

But developing these drugs is expensive and often a long slog – and what might look hopeful at an early stage of development cannot be assumed to be a guaranteed success in clinical terms further down the line.


So the lesson to us all from this week is let’s keep thinking big, but with our feet on the ground – even if, sometimes, media headlines tempt us to do otherwise.

Tuesday, 14 October 2014

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.

Thursday, 9 October 2014

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. 

Friday, 3 October 2014

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/.

Tuesday, 30 September 2014

Cystic Fibrosis Trust research sandpit on adolesence

Jessica Jones, Policy Adviser at the Cystic Fibrosis Trust, reports back from a 48-hour residential research 'sandpit' devoted to the subject of adolescence. Research sandpits are a key part of the Trust's research strategy, and bring together experts from within and outside cystic fibrosis to stimulate new thinking and innovation.

Last week I spent 48 hours holed up in a remote hotel with 24 top researchers, clinicians, specialist cystic fibrosis (CF) nurses, clinical psychologists, behavioural insight experts and CF community representatives; and not once did they tell me to do my physio!

I was a strange child – never wanting to be a famous pop star, instead I imagined notoriety being reached through being a famous research scientist who cured diseases. It was particularly exciting therefore to be asked to attend the Trust’s adolescent sandpit.

The innovative event drew together 25 experts from across the CF disciplines, and from as far afield as the United States; throwing together an eclectic mix of disciplines and asking them to leave their usual roles at the door. I was there to remind everyone of the patients in their clinics; the worries that may not be expressed; how much nebulising tobramycin tastes like paint stripper, and that people with cystic fibrosis have no more hours in the day than anyone else.

Challenges facing the participants included why treatment adherence drops off at adolescence and how to address this; how to accurately measure progress in changing bodies and the right kinds of support to offer patients and their families at this already challenging age.

Being a part of the event was a privilege. There was a constant buzz in the room as animated debates were held; coffee-fuelled discussions continued into the evening, and individuals were able to take a step back from the daily grind to take a fresh look at the issues at hand. Hopefully those 48 hours will have given time for the seeds of ideas to begin to germinate, and grow into something which will push back the decline in the health of those with cystic fibrosis, and stop adolescents having to worry about their futures.

Friday, 26 September 2014

Cystic fibrosis registries: behind the headlines

A study in BMJ Thorax has revealed that children and young people with cystic fibrosis in the UK  have poorer lung function than their peers in the US. Here Rebecca Cosgriff, Registry Lead at the Cystic Fibrosis Trust, explains why the study illustrates the power of clinical registries and collaborative working.

Today saw the publication of an important collaboration between researchers, which used cystic fibrosis (CF) registry data from both the UK and the USA. This marks a milestone in the increasing usefulness of patient healthcare registries.

There are cystic fibrosis registries running all over the world; including Europe, Australia and the United States. The UK CF Registry is funded and managed by the Cystic Fibrosis Trust; an indispensable research and quality improvement resource made possible by the generosity of donors to the charity.

All of these registries collect data on people with cystic fibrosis, tracking their care and associated health outcomes. However, because the healthcare system of each country is different, there is inevitable variation in the type of data held by each registry, as well as how it is collected. This UK and US data comparison marks the most comprehensive attempt to-date to meaningfully compare two different CF datasets. Complicated statistical methods and clinical expertise were needed to ensure that, as far as possible, this research compared apples with apples, not apples with pears!

Now that these methods have been worked out, we can start to detect differences between patient characteristics, models of care, and clinical outcomes. For me, the important part of this paper is not the fact that there is a three per cent difference in average FEV1% predicted between the US and the UK. Rather, it is remarkable that we now know that this difference exists, and can start to understand why that might be the case.

This demonstrates the power of clinical registries, and collaborative working, to influence medical practices for the better; enabling healthcare providers in the NHS to strive for excellence compared to their counterparts both nationally and, now, globally.

Friday, 5 September 2014

Let's Do Lunch!

All this week the Trust has been trying to raise awareness about nutrition for children with cystic fibrosis, especially as they all head back to school.

Mary Judd, Paediatric Respiratory Dietician at the Royal Brompton has put together this blog, including some ideas for how to mix it up a bit when packing your child's lunch.


Every child with cystic fibrosis has different nutritional needs and requirements. Generally speaking their nutritional requirements may be higher than their friends because they need extra calories and nutrients to help them fight infection and keep their lungs strong, particularly if they get sick with colds or the flu.
Lunch is an important meal as it helps children maintain their energy and concentration levels through the school afternoon. With the right balance of nutrition, extra fat and calories, and prescribed supplements, children, teens and adults alike can all help to keep themselves healthier.
Here are some ideas that our patients and their families have told us work well in lunch boxes, especially when you get bored of the traditional sandwich!

  • Think about pasta and pesto, salads or cheese and crackers to mix things up a bit.
  • Gone are the traditional days when people wanted to just have chocolate bars and crisps (although these are good too). Flapjacks or shortbread can have as many calories as a chocolate bar. Also, add Mini Cheddars, Twiglets, pastry and cheese straws.
  • Mix any wrap or potato filling with mayonnaise or olive oil to increase the calories – such as tinned tuna or salmon, egg, or chicken (sliced or left over roast).
  • Babybels, cheese strings or cheese triangles can be thrown into any lunch box as an extra.
  •  Dessert pots (Cadbury do different versions) or mini rice and custard pots. These are great because they tend to be high in calories. Don't forget to pop a spoon in!
  • Scone, malted fruit bread or hot cross bun with butter or cream cheese and jam.
  • Peanut butter can be tasty and high in calories but check the school’s policy on nuts.
  • Left over pizza or quiche.
  • Samosas.
  • Picnic plate – salami/ham, cheese cubes, crackers, dips, vegetable sticks.
  • Dried fruits – especially yoghurt or chocolate-covered.
  • Bagels (cinnamon and raisin or blueberry; with cream cheese works quite well too).
  • Fruit juice or yogurt-based smoothies are a great way to add some vitamins or minerals. Also milkshakes (Frijj, Nesquik or Yazoo) are usually welcome additions. However, the dietitian may sometimes recommend a higher calorie version of milkshake on prescription.