Friday 30 January 2015

Trials Week: Postcard from the European Clinical Trials Network conference in Barcelona

This week, Dr Janet Allen, Director of Research & Care at the Trust, has swopped frozen England for sunny Spain, and the European Clinical Trials Network conference as part of the Cystic Fibrosis Europe meeting. Here’s what’s been going on.

Every January, the research-active organisations across Europe that represent people with cystic fibrosis (so the Cystic Fibrosis Trust in the UK) come together to share experience and knowledge under the umbrella organisation known as Cystic Fibrosis Europe (CFE).  At the same time, we meet with colleagues in the European Clinical Trials Network or ECTN. This network was set up by CF physicians to coordinate clinical trials across selected centres in Europe. By working together, we can increase the number of trials running in Europe and attract international global companies to bring their clinical studies to Europe.  

It’s the end of January, so where better to have a meeting than in Barcelona, or more accurately a town just outside Barcelona:  to swap cloudy, windy, cold Bromley for blue skies and a slightly warmer environment! However, before anyone gets carried away, I can say we have been stuck in a stuffy room all day and the blue skies have not been seen as we debate methods to increase access to clinical trials, standardisation of protocols and better ways of getting protocols adopted through many different languages. The commitment and passion of the ECTN members to improving access to clinical trials across Europe is remarkable, especially when I realise that all the discussions are in English, even though for the vast majority, English is not their native language.

The ECTN has been running for a number of years and initially involved 25 centres across Europe; five of which are in the UK – Belfast, Birmingham, Royal Brompton, Leeds and Nottingham. The Cystic Fibrosis Trust provided financial support to help form the ECTN over the last three years.  

The ECTN has been very successful and there are now plans to expand the number of participating centres by 10. They have been tracking the number of trials and can show an increase over the last few years. In addition, the ECTN has been instrumental in assisting in trial design and doing feasibility studies. 

Thursday 29 January 2015

Trials Week: Clinical Trials: clearing up some common misconceptions…

As part of our week raising awareness about clinical trials in partnership with CF/Aware, we asked our very own Senior Research Manager, Dr Anoushka de Almeida-Carragher, to look at two of the key myths around trials.

Clinical trials have a profound impact on our lives, and there are a couple of myths associated with them that are worth clearing up.

Myth: “A clinical trial, which has shown a particular drug to be ineffective, is a failure
A completed clinical trial that has shown a particular drug to be ineffective should not necessarily be regarded as “unsuccessful”, and therefore a failure. 

Quite often, the drug just wasn’t effective enough or suitable for the purpose for which it was tried, under the conditions tested, and purely knowing this , is, in itself, very informative. In fact, the drug may actually be beneficial in other ways. A classic example is Viagra, which was initially tested as a treatment for angina but the trial results were disappointing. Pfizer was about to pull the plug on this “unsuccessful” trial, when it was reported that it had a surprising alternative function… Viagra is now one of the most commonly prescribed drugs, globally.

So, if a trial doesn’t show the results we want to see, it doesn’t mean it is a failure. In the world of medical research, acquiring knowledge, even from negative results, is invaluable. As Thomas Edison once said, “I have not failed; I’ve just found 10,000 ways that won’t work”.

Myth: “A Clinical trial testing a brand new drug is more important than one studying an existing drug.”
Of the two main types of clinical trial, one may test a new drug, while the other might look at a new combination of existing treatments, or test whether administering a known drug in a different way can increase its efficacy and/or reduce its side effects. When it comes to patients signing up for clinical trials, some patients are put off based on the type of trial being carried out. For instance, a clinical trial testing a shiny new drug may seem more appealing, purely for the possibility of a new life-changing intervention, whereas any other type of trial involving existing treatments would not have that air of novelty and anticipation. 

What we must not overlook is that trials of this type are also invaluable, especially those where comparisons are made between existing treatments, like the current TORPEDO trial. This trial compares the administration of two existing treatments of the Pseudomonas aeruginosa lung infection in people with cystic fibrosis, (ie it compares intravenous vs oral antibiotics). This is a good example of a trial which doesn’t involve a brand new drug but which, once completed, will give us the valuable info we need to decide which method of administration works better, and is more beneficial to the CF patient in the long run.

It is vital, therefore, not to discriminate between the different types of trial; all clinical trials, irrespective of the category they fall in to, are equally important.

You can see more about Trials Week by following the hashtag #trialsweek on Twitter.

Wednesday 28 January 2015

Trials Week: The Cochrane Collaboration – Evidence and practice

It may surprise you to learn that what takes place in the lab or on the ward is only the tip of the iceberg. As part of our joint Clinical Trials Week, today we and our friends at cf/Aware are each offering an insight into the vital work that goes on elsewhere to inform and sometimes form the basis for, a clinical research project.

Here Nikki Jahnke, Managing Editor of Cochrane Cystic Fibrosis & Genetic Disorders Group, talks about the work of the Cochrane Review. 

There are thousands of clinical trials being run across the world, so how can your doctor keep up with all their findings and make sure you get the best treatment? It would mean reading hundreds of articles in so many journals that there wouldn’t be any time to see patients! This is why we work to include the findings from randomised controlled trials - the best type of trial design for establishing whether a treatment works or not – in systematic reviews.

Why are they called systematic reviews? Because we work systematically through databases and journals to make sure we capture all the available evidence for a particular topic, like ‘inhaled antibiotics for pulmonary exacerbations’. Once we identify the relevant trials, two independent authors record all the information they need from the trial reports and collate it in a single review. By combining data from a number of similar trials we can be more confident that our findings really are due to the effects of the treatment and not just chance. Our reviews also consider if there are factors that might affect the results of the trials. We make recommendations for doctors and other healthcare workers to use in daily practice, which can inform patients when discussing treatment options with their doctors.

If we don’t find any trials that help us answer our research question, we add a plan to our review describing how we think trials should be designed to provide us with the information we need. Many agencies ask for a systematic review to be undertaken before they agree to fund new trials. The review will show if the trial needs to be run, ie because there is either no or little evidence, and indicate how it should be designed. An example of this is the review looking at antibiotic strategies to eradicate infection with Pseudomonas aeruginosa in people with cystic fibrosis. Our original review found some trials, but the findings were not conclusive. So we recommended that a new trial should be run with more people. The TORPEDO trial is now underway and when it is finished we will be able to update our review with the results.

Finally, how do we decide on what questions to ask and which topics to investigate? For this we rely partly on our volunteer authors (doctors and other healthcare workers), but we also ask people who have CF to tell us what questions they want answered. This is where you can really get involved in driving research that will directly affect you. Take a look at the 75 CF reviews we have published so far and let us know what we should look at next.
Visit cf/Aware's Facebook page this afternoon to hear from Rebecca Cosgriff, Registry Lead at the Cystic Fibrosis Trust, about the work of the UK CF Registry.

Monday 26 January 2015

Trials Week: An introduction to clinical trials

All this week the Cystic Fibrosis Trust and cf/Aware are joining forces to highlight the importance of clinical trials and why it’s vital for people with cystic fibrosis to get involved where they can. To get the ball rolling, Professor Alan Smythof the University of Nottingham, explains exactly what a clinical trial is and how it works.

A clinical trial is a fair test of a treatment. This treatment can be compared with a dummy treatment (placebo) or an alternative treatment (eg another similar drug). The results tell us if the treatment is effective and whether any harmful effects are seen. Clinical trials have brought about effective treatment for a range of conditions, which used to be killers, such as tuberculosis and leukaemia. More recently, clinical trials have shown that a new drug (ivacaftor) is effective in some people with cystic fibrosis (CF). Clinical trials must have a minimum number of patients. If there are too few patients then we may miss a real difference with the treatment we are testing. Drugs may work differently in children and adults, so we need to do clinical trials in both age groups.

Doctors know that taking part in a trial is a big ask for patients. Extra visits or additional tests may be needed – though we keep these to a minimum. However, we must do these trials if we want to improve the treatment of cystic fibrosis. There are many questions which we still need to answer - from evaluating exciting new treatments like ivacaftor, to testing which antibiotic works best for lung infections.

TORPEDO-CF ( is an important cystic fibrosis trial which is recruiting at the minute. TORPEDO-CF compares intravenous and oral antibiotics to see which is most effective in getting rid of early lung infection with Pseudomonas. Patients in both arms of the trial also get a nebulised antibiotic. The trial is important because Pseudomonas has a huge effect on patients’ lives, if it becomes chronic, and because intravenous treatment is a much bigger hassle than oral antibiotics. To see which treatment is best, the trial must recruit 280 patients. So far we have almost three quarters of this total. With your help we can finish the trial!

Our friends at cf/Aware have produced a glossary of clinical trial terms, to clear up some of the more complicated terminology.

Thursday 22 January 2015

'Designer babies' and cystic fibrosis: Where's the connection?

Our Director of Research and Care, Dr Janet Allen, takes us through the recent discussion around 'designer babies' and where cystic fibrosis fits in.

The recent media focus on ‘designer babies’ has challenged thinking about what the future could hold; using tools to correct mutations in genes such as those that cause cystic fibrosis. 

It is important to understand that the correction of mutations (also known as genetic editing) can be done in the laboratory today using CRISPR technology. In fact the Trust is already funding two programmes in this area for cystic fibrosis. At the same time, a small biotechnology company has been pioneering a slightly different approach of repair called RNA editing and is about to start on very early clinical studies. 

Gene or RNA editing are both forms of gene therapy, although different from conventional gene therapy which we have been funding through the Gene Therapy Consortium. Conventional gene therapy inserts the corrected gene into the airway cells; in the editing approach, the mutation in the person’s own gene or RNA is corrected. The big question is whether it is possible to transfer the laboratory successes and correct CF-causing mutations in people’s lungs.

These are early days and already people are thinking about the next, next generation of gene therapy, combining genetic editing with stem cells to overcome constraints on delivering the corrected gene. It is possible to take cells from a person and, in the laboratory, convert them into stem cells (called iPS) specific to that individual. The CF-causing mutation can then be edited in the laboratory and the stem cells converted back to lung cells. A Trust-funded project is just about ready to start and we should know by the end of this year whether it has been successful in the laboratory.

So what does this all mean for the designer baby debate? Well the technology to edit out the CF causing mutation is exactly the same for designer babies as the next generation and next, next generation gene therapy approaches already taking place. 

For designer babies, this editing would need to be completed in the embryo and this is where the ethical considerations become the overriding issue and it is good that we’ve been challenged to start the debate, well before any of this advances. The debate gets tricky because the same technology that could correct for conditions such as cystic fibrosis could be used for non-health issues, such as hair or skin colour that a parent deems less desirable.

In the UK, we have an excellent framework to control research activity and ensure topics like these are well-thought through and all ethical considerations given time. Legally, this area is tightly controlled by the Human Fertilisation and Embryology Act 1990. Pioneers in the UK such as Dame Mary Warnock developed the thinking and ethics that allowed IVF to start and the recent ‘three parent’ programmes for some muscular dystrophies. In many respects, through the formation of the Human Fertilisation and Embryology Authority, these pioneers have provided the framework to safely explore challenging scientific advances such as designer babies and for the UK to lead the debate and provide thought-leadership in this area. It is good to hear also that the highly respected Nuffield Council on Bioethics is also considering the matter.  

Wednesday 21 January 2015

Lung function in children with cystic fibrosis: A comparison between the UK and the US

Dr Anoushka de Almeida-Carragher, Senior Research Manager at the Trust looks at the recent discussion around the differences in average lung function between CF children here in the UK and those in the US.

In September last year, a paper was published in the journal ‘Thorax’, which reported that the average lung function in children and young adults with cystic fibrosis (CF) in the US was significantly better compared with those in the UK. The study was conducted by Christopher Goss and colleagues, based in the US and the UK. They collected data from the UK and US Registries and compared CF outcomes and the use of treatments.

The results of this study have undoubtedly raised some concerns, especially among some CF researchers in the UK. One issue is the fact that the UK Registry is more representative of the whole UK CF population (who have universal access to care via the NHS), whereas the US’s Registry only captures data of patients attending accredited CF centres. As a consequence, the UK’s data may show info from more disadvantaged socio-economic groups resulting in poorer clinical outcomes, compared with the US.

The paper does not explore the reasons, in great depth, behind the difference in lung function, but it does suggest that in the UK, chronic pulmonary therapies (like sterile hypertonic saline solution which helps clear mucus from the lungs) had been used much less frequently to treat young people compared to the US. They also speculate that the difference could be due to the difference in frequency of visits to CF centres; in the US, CF is only treated at specialist centres, while in the UK, care is managed between local hospitals and specialised units, and in some parts of the UK, young patients may only visit a specialist centre as little as once a year and then visit their local hospital the rest of the time, where they may not be seen by a CF practitioner.

What we can conclude is that making comparisons between one country and another is useful and informative, especially where there are obvious differences in care and treatment methods. Even though the results of this study show a lung function discrepancy between the two countries, the explanations for such differences are not definitive. Further investigations, such as cross-country longitudinal studies using Registry data, on a like-for-like basis, are essential before we can draw conclusions between treatments and outcomes in the global CF population.

You can learn more about research into cystic fibrosis at

Saturday 17 January 2015

Remembering Emily Thackray

Emily Thackray was a committed and enthusiastic campaigner and fundraiser for the Cystic Fibrosis Trust for many years, and sat at the very heart of the community throughout her life. Emily died on 28 December 2014 and today she will be remembered and celebrated at her funeral and wake.

This blog was written by Oli Lewington, Engagement Director at the Cystic Fibrosis Trust – a friend of Emily’s – in the days following her passing.

Lots of things will be written and spoken of Emily Thackray in the next few days and weeks. She died yesterday after a second double-lung transplant proved too much for her body to withstand.

Emily’s unique ability – using unique in its literal sense, as I’ve never come across anyone with the same gift – was to make everyone she ever came into contact with feel like they were the most important in her world.

There are dozens of people who will be grieving the loss of a best friend today, because that’s who she was to everyone: selflessly sharing her love and compassion for the world with all she brushed against and, in the process, making everyone she touched feel special, feel like they mattered. She made a difference.

Equally, everyone who knew her will have their own ‘Emily’ with whom they spent time, shared laughs and cried when it was warranted. We all knew a different friend who gave different things to our lives.

My Emily came into my life in the early days of the internet when I first discovered the Cystic Fibrosis Trust forums: she was already there and dispensing support and advice as needed. I struck up a friendship with her and with some of the other frequent posters and we supported each other through tough times of losing friends that we were terribly close to. It seems nothing much changes in a life with cystic fibrosis.

When she set up the organ donation campaign (now charity), Live Life Then Give Life, with her great friend Emma after the loss of more than one mutual friend on the waiting list for transplant, I offered to help in any way I could. I ended up being one of the first Trustees of the charity and being part of the team that one Best Campaign Team at the 2008 Charity Times Awards and Best New Charity the following year.

My Emily was always one step ahead of me on my CF journey. She was the first of us to start needing supplementary oxygen. She was the first to use a wheelchair. She was the first to have a lung collapse. She was the first to be assessed for transplant and, thank God, the first to receive it. She was the first to be married after her transplant, and the first to have serious complications. Now, she’s the first of the two of us to go.

What became indelibly unique, though, was that everything she went through became a source of help and information for others. She never hid away from anything and always used her own lived experience to make it even a tiny bit easier for others going through it. She supported me as I took every step and misstep she took, a few months further down the line.

The day I finally got my transplant call I remember sending her a message and getting an immediate phone call back.

Take some paracetamol now,” she told me.

I wasn’t sure if I should, but she countered immediately, “The stress of the situation might raise your temp and if it does they won’t go ahead. Take two paracetamol now and it will drop your temp if you have one, but it won’t mask anything more serious that could be a real contraindication.”

I took them. I passed the tests. I got new lungs.

The story that sums up Emily, though, came through on my Facebook last night from one of my oldest friends and was one that I’d never heard before. This stands as testament not only to her willingness to help and support anyone and everyone, but also to be humble and quiet in going about it.

She was so wonderful when you got your call, patiently, calmly keeping me informed about the stages, what to expect, what were the good signs, what to worry about & what to cheer.

All the way through your surgery and recovery she stayed in touch, answered my many emails and sent me random messages asking how I was doing – she had volunteered herself to essentially be my support as I didn’t want to bother your parents or K too much with my need for information and updates. It meant such a lot to me and I was incredibly appreciative knowing she was a message away to answer a question or calm a worry.”

That’s Emily: friendly, warm, generous and patient. And not just my Emily, that’s everyone’s Emily.

Em, you will be missed far more than most of us can understand, but we remain ever grateful for the joy and happiness you brought to our lives, for the connections and friendships you forged that will last long into the future, and for the blessing of finally understanding one of my favourite quotes:

She was a line of poetry in a world of prose.”Polly Toynbee

Thispost first appeared on the SmileThroughIt blog. If you would like to make a donation to the Trust in Emily’s memory, please click here.

Wednesday 14 January 2015

New Medicines in Scotland

On Monday, Cayston, a new nebulised treatment to tackle Pseudomonas in people cystic fibrosis was approved for use in Scotland. Here, Yvonne Hughes, Public Affairs Officer for Scotland tells us more about how such decisions happen.

The decision this week by the Scottish Medicines Consortium (SMC) to approve another drug for cystic fibrosis underlines how the work the Cystic Fibrosis Trust does here in Scotland is making an impact. As Public Affairs Officer, ensuring access to new medicines is a part of my job that may appear a bit boring at first, but is an essential and enjoyable part of my work and allows the Trust to make the representation on behalf of the CF community.

With the help of clinical CF staff across Scotland, I am able to contact patients who are on the trial for the new drugs being assessed. It is crucial that the patient experience and that of their families is captured in papers we submit to the SMC as it could be the difference between a Yes and a No recommendation. Without your help drugs like Kalydeco and Cayston may never have got to clinics, so I am extremely grateful. I have also made a few friends in the process! As someone with cystic fibrosis I understand how isolating it is, so to speak to you and hear your stories is always a pleasure.

The recent Cayston decision fell under changes made last year to the way medicines are appraised to improve access for those at end-of-life or with a rare disease. A Patient and Clinician Engagement meeting (PACE) has been introduced where it is likely a drug may not be approved. Cayston went through this process and we found it to be valuable, particularly as we were able to talk about living with cystic fibrosis direct to people involved in making decisions around new medicines.

The changes to the SMC will be up for review this year and I shall feed in to that process but in the meantime I will continue to ensure that patients with cystic fibrosis are represented when new drugs come online. Please feel free to leave a comment or get in touch with me

Monday 5 January 2015

Newborn Screening

With newborn screening in the news, Rebecca Cosgriff, UK CF Registry Lead, looks at the difference the heel-prick has made in detecting cystic fibrosis sooner. 

2015 is off to a great start with the news that newborn screening in England will now detect more genetic diseases.  Wales will expand its newborn screening programme later this month, whilst Scotland and Northern Ireland are yet to announce a decision.

Newborn screening involves pricking the heel of a baby between five and eight days old, so that the blood sample can be tested for genetic disorders. It is important to diagnose genetic diseases as soon as possible, so that treatment can begin before organs are damaged.

Following a Cystic Fibrosis Trust campaign, newborn screening has been routinely used to detect cystic fibrosis since 2007. UK CF Registry data shows that since then the median age of diagnosis each year has dropped from five months to 30 days. The number of people diagnosed each year by the time they are three months old has increased from 46% in 2007 to 72% in 2013. During the same period the median age of death has risen from 24 to 29 years.

It’s fantastic that the example of cystic fibrosis, along with other genetic diseases already part of the programme, has paved the way for similar improvements in these new areas.