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.

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.

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.

Transformational Treatments in Cystic Fibrosis

Today is the UK CF Conference, Britain's largest event dedicated to multidisciplinary cystic fibrosis work. We were honoured this morning to have Preston W. Campbell III, MD, Executive Vice President for Medical Affairs at the Cystic Fibrosis Foundation (our US counterpart) deliver a keynote on transformational treatments. Below, Dr Campbell gives more insight into this groundbreaking work.

It is an exciting time in cystic fibrosis. We have achieved remarkable improvements in the health and survival of those living with CF in the U.S. and U.K. over the last several decades, primarily due to advancements in CF care and the development of new therapies targeting the symptoms of CF. 


By the late 1990s, we had learned a great deal about the recently discovered CF protein, CFTR, but that information had not been translated into new CFTR-based therapies. To spur the development of new treatments, the CF Foundation began a new program focused on applying new drug discovery technology to treating the basic defect—the faulty CFTR protein—and funded a number of novel research collaborations. 

Vertex was successful in identifying small molecule compounds that targeted CFTR, and in 2012, ivacaftor was approved for patients with the G551D mutation after clinical trials demonstrated that it was safe and had remarkable clinical benefits. 

It is likely that ivacaftor will treat 15% of CF patients as it is approved for other CF patient groups. The most common mutation, F508del, has an additional problem and requires a different approach to treat effectively. 

Recently, a Phase 3 clinical trial was completed in patients with two copies of F508del, who were treated with ivacaftor in combination with another potential therapy, lumacaftor. Patients on the combination drug experienced improvements in lung function, reduction in pulmonary exacerbations and improved weight gain. 

This combination therapy will now be submitted to regulatory agencies for approval for patients with two copies of F508del (50% of patients). Another corrector is being evaluated for patients with only one F508del mutation. 

In addition, a robust new screening effort has been underway for over three years that will improve the efficacy of treatment for those with one or two F508del mutations. New screens are also ongoing for those with rare mutations, including nonsense mutations. If these programs are successful, 98% of CF patients could potentially be treated with these transformational therapies. The remaining 2% of the CF population will need other approaches, such as gene therapy, that replace CFTR or strategies that bypass the defective protein. These potential new therapies hold great promise for the CF community, and I am proud to be a part of this effort.