Monday, 29 April 2013

Speech to CF Trust Scientific Conference 2013

29 April 2013 at The Wellcome Trust, London.

"Good evening, everyone. It’s an honour to be able to speak to you tonight. It’s a bit overwhelming actually. I feel a bit like a kid speaking to a room full of Father Christmases.

I want you all to imagine – you can close your eyes if you like - I want each of you to imagine you’re in a little wooden boat floating in the middle of the ocean. As you bob up and down, you feel the sun on your back, you can hear the distant sound of birds and you can smell the sea air. Sounds pretty nice. But there’s a problem. The boat is full of holes and water is gushing in. There are sharks. All you have (to bale out the water) is a tea spoon. You spend most of your time baling with your tea spoon trying to stay afloat. You do it all day. You go to sleep, you wake up in a boat full of water and you start baling again. It is exhausting and relentless. You need a bigger spoon. Better yet, you need to find a way to plug the holes. That’s what CF feels like to me.  It’s a fight against drowning in my own mucus.

I was diagnosed in 1978 at the age of 3. When I was 14 my consultant told me I’d probably need a heart and lung transplant in my early twenties and that I’d be lucky to live beyond 25. This [Spoon 1] was standard of care in those days. Well my heart and lungs are still original and I’m still here at 37. During that time, we’ve seen the development of better spoons - nebulised antibiotics like TOBI [Spoon 2], mucus busting therapies like Pulmozyme and Hypertonic Saline; and more effective airway clearance techniques [Spoon 3]. These all resulted from research and I might not be here without them. I want this [Spoon 4] but I have this [Spoon 3]. Kalydeco is a bit different – it’s plugging holes - but I’ll come on to that.

New treatments are exciting and important but, actually, I think it’s just as important to develop better tools to manage symptoms and figure out how to use the tools we already have in a more effective way. 

So I want to talk about three things: the therapeutic portfolio, clinical trials and adherence.


Vertex’s new drug Kalydeco is a small molecule with big implications. This little blue pill is changing the lives of the 4% of CF patients with the G55-1D mutation. Trials to see if Kalydeco works for other similar mutations are underway. Phase 2 data released by Vertex on April 18th, in a press release, has bolstered hopes that Kalydeco might work in combination with various other small molecules to treat a much broader range of CF patients including people like me with the most common delta F508 mutation.

This is incredible – when I read the blogs and hear the stories of people on Kalydeco – it is mind-blowing. It brings a tear to my eye. But there’s a long way to go from Kalydeco to a Kalydeco-like therapy for all 1,800 mutations and it is important to remember that, even for G55-1Ds, Kalydeco is not a cure. These people still have CF.

They still need to do all their other treatments. They still have chronic infections and exacerbations that mean they need IVs - but not as often. They are significantly better, able to do more stuff without getting breathless, put on proper weight, bounce back faster from exacerbations and generally far more resilient. Those on Kalydeco seem able to shake off a cold without it turning into an exacerbation – in other words two weeks in hospital becomes 2 days off work. I hear about patients with G55-1D who’ve come off the transplant list or who no longer need a wheelchair or oxygen therapy. They can go back to work or perhaps go on holiday. Maybe their parents no longer need to act as such full-time carers so they can go back to work or have a more normal life too. These patients are able to think about and plan for the future in an entirely new way. The trajectory of their disease has changed. It is hard to explain because the way CF affects patients and families is so complex - and maybe we need to find better ways of articulating the true value of these medicines - but this is a profound leap forward and it is exciting to think the same approach may help those with other mutations.

For young children whose lungs and other organs are not yet damaged, drugs like Kalydeco could be very close to a cure. For older ones whose organs have been damaged, Kalydeco might freeze the disease but it won’t reverse damage already done. So even if there was a Kalydeco-like treatment for all mutations, symptom-management tools will still be needed. In fact, they may well be needed more than ever because people with CF will be living longer. 

CF is a complex disease and it seems likely that complex combinations of small molecules and other kinds of therapies will be needed. Lots of money is going into small molecule CFTR modulators. As a patient, I worry we are putting all our eggs in one basket. It may not work. It may half work. I think it’s vital for the CF community to nurture some alternative approaches and facilitate some alternative science. I think we need to hedge our bets.

The CF Trust has always been committed to research and I think this commitment is more important now than ever before. Of course, the Trust established and funded the Gene Therapy Consortium and this is pioneering new techniques. While even this is not a “cure” as such it may turn out to be just as important as small molecules or more important. This would not have been possible without the Trust’s funding and the tenacity of those involved. I hope the Trust can continue to support this programme as part of a broader portfolio.

It’s essential that the CF Trust works in conjunction with the CF Foundation and others to achieve a diversified portfolio of therapeutic options. We need to go after better spoons - more effective symptom-management tools (particularly those which can reduce inflammation or neutralise Pseudomonas and other lethal bugs) – as well as looking for new ways to rescue chloride channel function - to plug the holes.


In the UK, about 10% of CF patients have participated in a clinical trial. We need to do more. We make it far too difficult for sponsors to do trials. We need to make it easier because we’re all losing out.

Studies show that simply taking part in a trial can improve a patient’s health and that is irrespective of whether they receive the active drug or placebo; and irrespective of whether the trial has a positive or a negative result. It is not just about being an altruistic guinea pig, it’s about direct benefits (for patients, clinicians and hospitals) and establishing a more positive culture that forces everyone to raise their game and not to settle.

This means making sure that, when there is a new drug, our patients have the opportunity to take part in clinical trials but it also means doing more trials to make sure we are using the tools we already have as effectively as we can.

Trials, funded by the CF Trust, like the TOPIC study on once vs three times a day aminoglycosides, have resulted in significant improvements in care. In some cases they have allowed treatments to be simplified and ineffective ones to be stopped. These kinds of trials are really important and we need to do more.

It also makes sense to get patients and parents involved in clinical research at the design stage. After all, clinicians only see patients and collect data when the patient comes to the clinic - or in hospital. It is the patients and their families who live with CF day-in/day-out. We are the only ones with a 360 degree view. Why can’t we use this experience, and data collected at home, systematically to inform clinical trials, and clinical practice more generally, to make sure we’re asking the right questions and working towards the outcomes that are most relevant to patients in their daily lives? Internet technology has made this easy to do and, if our aim is to improve real world outcomes, I think we have no choice but to embrace it. That’s why I’m really excited by initiatives like CFUnite (backed by The Wellcome Trust), Cochrane and the James Lind Alliance; and the CF Trust’s commitment today to do more to facilitate patient involvement in this more strategic aspect of clinical trials.   


This is a big deal and we need to talk about it more. One of the most challenging aspects of having CF, for me, is the heavy burden of daily treatment. Every day I take about 40 pills, 7 nebulisers, a saline nasal rinse, 4 inhalers and do 2 or 3 injections, blood sugar tests and two sessions of self-administered physio. I spend a lot of time washing and sterilising nebuliser equipment. All this takes 3 hours on a good day and up to 4 hours when I am less well. This is my normal. When I get an exacerbation I typically need a 14 day course of hospitalised IV antibiotics. This tends to happen 2 or 3 times a year. I am lucky, for many it is much worse.

So CF adherence is not just popping pills - this is real work, it is difficult and it can be exhausting. You can never take a day off. Some of the treatments are unpleasant - they require a lot of effort and willpower. It becomes much harder to get all your treatments done, and it takes longer, when you’re not feeling so well which is, of course, exactly when you need them most. It is easy to feel overwhelmed with treatments. It’s hard to find a balance. It is hard to create reliable windows for work, family and friends; and for all the other things you have to do to get by in life. 

So we need to find ways to reduce the burden of treatment. Something which saves an hour a day and can be done anywhere is going to have a dramatic impact on quality of life and, I think, adherence. Clearly there is a link between poor adherence and hospitalisation. We do too much fire-fighting and we need to focus more on prevention. We need to recognise the value of prevention.

It may mean simplifying treatments; developing a pill or inhaler to replace a nebuliser (more things like Bronchitol, TOBI Podhaler and Colobreathe); or more creative things, perhaps bringing in some sports psychology techniques; or web-based platforms or smartphone apps that help to manage and motivate or allow people to track their progress or even just talk to someone who’s in the same situation (because don’t forget we can’t meet up due to the risk of cross-infection). This kind of thing could help people live a fuller life, stay healthier, reduce exacerbations and keep them out of hospital. This is worth doing, big time, and I think it’s great that the Trust has highlighted adherence and reducing the overall burden of treatment as key research priorities. It’s a simple relation: reduce the burden, improve adherence; reduce exacerbations.

So here’s what I want you to take away:

The CF Foundation and Vertex have blazed a regulatory trail, given us new tools, shown Pharma that CF is a real market, shown everyone what can be achieved when Pharma works with a patient group. They have delivered a transformative drug for a very small sub-population of CF. I see this as injecting oxygen into the CF research environment. It’s woken people up. It’s like our Big Bang. We need to capitalise. We need to use it to our advantage. That’s why the Trust’s new strategy launch is perfectly timed. It provides a great scientific map but it also sends a clear signal of the Trust’s intent to collaborate with new partners, in new ways and to make resources available to help everyone in the CF community convert their ideas into new therapeutic tools.

And as Yoda said: “do or do not, there is no try".

Thank you." 


  1. As always Oli, you have hit the nail on the head. Fantastic speech - wish I could have heard it first hand - but I'm sure it went down well. I really hope the right people were listening and you don't just get spoon 4, but rather a great big shovel!!

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