People with cystic fibrosis take matters into their own hands by playing an active part in clinical trials.
Rob has been taking part in scientific studies since he was seven years old, when he began attending the specialist CF clinic at the Royal Brompton Hospital. His mother, Alison, remembers that participation then was relatively quick and low-level, done during Rob’s routine visits to the clinic, like “sticking something up Robert’s nose just to collect something. It was always that kind of thing.”
“I was in a very research-heavy clinic, so I was exposed to a lot of it from a younger age, and I just thought it was normal,” says Rob. “I remember doing one where they put a bit of sugar in my nose and timed how long it was until I could taste it on the back of my tongue… I’ve since worked out it was because they were looking at how well the cilia work in my nose. I think they did tell me at the time, but I didn’t know what cilia were, and I just remember thinking ‘Oh yeah, sure, I’ll do whatever this thing is, it sounds easy’.”
As an adult, Rob has continued to participate in clinical studies when he has the time. He is not alone in his active interest in CF research – Rob is one of a large community of people with CF who track the latest research developments online and follow the fortunes of the latest clinical trials.
“Duty feels like a strong word, but it is something like that… It’s not like anyone in the world can go and do these things,” he says. “The whole reason they need people like me is because I have CF… and [there’s] a smaller pool of those people to test things on. I know that some of them either might not work or are so long-term that I’m unlikely to see [the benefits] in my lifetime. But… having a reasonably scientific background [Rob has a degree in maths and statistics], if they explain to me why they’re doing something or what their aim is, that’s usually enough for me to say this might be good for me [or] it might work for someone else in the future.”
A medical treatment or cure is not just the culmination of years of dedicated research. It is also the consequence of many volunteers’ donated hours, sat in hospitals, testing drugs, having tubes stuck up their noses. “It can take quite a lot of time, so it’s a case of balancing it… with work,” says Nick, a 38-year-old businessman in London. Like Rob, Nick has CF and an active interest in research. “On some trials, you don’t know quite what the side-effects will be until they get to a more advanced stage. I’ve never had any permanent side-effects, but sometimes… you’d have reduced lung function or just feel really pretty dreadful for two or three weeks… Don’t get me wrong, I’ve never felt that I have been in any kind of danger, [but it isn’t] always a particularly pleasant thing to do.”
Nick has a mild form of CF and lives a very active life, numbering diving and mountaineering among his pastimes, when he is not working as a director at the Royal Institution of Chartered Surveyors. “I have been slightly lucky in that, whilst CF has been a major irritation, the treatments that I have had are relatively effective, so I’ve been able to have a fitter life than most people.” This has spurred Nick on to volunteer for medical CF studies. “For me, it has been about trying to have an impact for all people with CF… It’s something I felt I should do… but I never really expected to get any direct benefit from the trials I participated in.”
Rob’s and Nick’s many years of participation in research mean they have been involved, one way or another, in many stages of the work that have led to the world’s largest trial of gene therapy in CF. But as this trial was beginning to get underway, Nick was faced with a dilemma. Fully committed to research and his CF community, he was hesitant when he was separately offered ivacaftor, a new wonder drug from California that specifically targets his type of CFTR mutation. Taking the new drug, which had been discussed worldwide with much excitement in CF internet forums, would mean that Nick would be unable to participate in the gene therapy trial. Remarkably, he offered to delay taking the new compound so that he could still enrol in the research, but the gene therapy scientists told him to go ahead.
Nick is still looking for ways to raise money and awareness for CF research. He was ready to climb Everest this spring, but called off his attempt following April’s extreme avalanche. He is optimistic about future treatments that could help a wider range of people with CF, and wants to see them developed sooner. “I think there’s an awful lot of hope, but I guess the key thing is really time… The faster the research can happen and you get through all the administrative hoops, the faster people can be helped. The longer it takes, the more people are going to die, to put it brutally.”
This feeling of urgency is shared by Bea, a 38-year-old with CF, who last year trekked part way up Mount Kilimanjaro to raise £5,000 for the Cystic Fibrosis Trust. “Everyone in the CF community sees this as buying our cure. [If we throw] money at it, we will definitely get somewhere.” This is a message that Bea is passionate about, leaning over the table and issuing the command “Donate!” directly into my dictaphone. “I really believe, hand on heart, that we’re going to get this licked in ten years.”