Jeremy, a trainee surveyor, plays football most weekends, often meets his mates in the pub, but is careful about what he drinks. He spends 45 minutes every morning and evening doing breathing exercises to help his lung function and consumes at least eight capsules of enzymes with every meal. His life at home is filled with as much activity as possible so that no precious moment is wasted.
Jeremy, 22, has cystic fibrosis (CF). The average life expectancy for a CF sufferer is 31.
I met Jeremy at King’s College Hospital. He had already spent eight days inside and was due home the next day. He was bored and lonely. Most of each day was spent in a room on his own, linked up to intravenous pumps, watching television, studying or playing computer games. He could not spend time with the similarly-aged man in the next room, Peter, because he also has CF and the danger of cross-infection is too great. In hospital, Jeremy has learnt how to administer the insulin he needs, as his pancreas has been damaged by the effects of CF and no longer functions properly. He has to have a spell in hospital like this around once a year.
At King’s I also met nine-year-old Helen, who had been enjoying an afternoon of art in the hospital school when we disturbed her. She was matter of fact about her exercises and enzyme capsules. She said she sometimes has a plastic port in her side so that medication can be administered intravenously. When this happens, she is not allowed to do PE or to play outside and this, she says, makes her “fed up”.
I was taken to see Jeremy, Peter and Helen by Dr Hilary Wyatt, a consultant specialising in CF in both children and adults. The CF Trust has campaigned with some success over a number of years for more centres of excellence such as King’s to be set up. Wyatt explained how important it is for any CF sufferer to be seen by a team of specialists, including doctors, nurses, physiotherapists, dietitians and psychologists, at least once a year to monitor their condition and provide treatment beyond that which may be available locally.
The unremitting nature of CF is clear. It is a genetic disease that affects a number of organs in the body (especially the lungs and pancreas) by clogging them with thick, sticky mucus. As a result, CF sufferers typically have problems absorbing nutrients from food, which explains Jeremy’s enzyme capsules and slight stature. They are also very prone to chest infections, which is why Jeremy does daily exercises. The effect of CF on the affected organs is cumulative and therefore increasingly debilitating as time goes on.
My day began with a two-hour talk on gene therapy from Professor Eric Alton at the Brompton Hospital. Alton managed to make his immensely technical area of expertise accessible even to a complete non-scientist such as myself. Gene therapy research, funded principally by the CF Trust, is carried out by a team of some 50 scientists across three sites in the UK. This structure is unusual, as most medical research is carried out by small teams on one site, often in competition with others.
The team’s research is at a crucial point. Clinical trials for a therapy that may add enough healthy DNA to sufficient of the cells lining the airways to relieve symptoms in CF sufferers are due to begin in less than two years. If the symptoms can be relieved in this way, then the relentless onslaught of the condition would at least be slowed, if not halted.
The raw materials for the trials alone will cost more than £3m, and when staffing and other costs are added, the total bill will be around £4.5m. Much of this funding will come from the CF Trust. The support of organisations that can provide substantial funds for a number of years obviously makes a big difference to the trust. Such support means it can guarantee funds over the timescale needed to make the scientific advances that can change the lives of thousands of CF sufferers.
This research carries with it so many hopes and possibilities, particularly for the families of young children whose organs will not yet have been irrevocably damaged by the condition and who may live longer and less-disrupted lives if the research that Alton is leading ultimately proves to be successful.
To add a legal, and literary, slant to a day where my background was irrelevant, CF sufferers are like the wards of court in Bleak House’s Jarndyce and Jarndyce: they should not live their lives in the hope of an imminent development, but if it comes it will change their futures immeasurably. Unlike the lawyers in Jarndyce, we can all make a real difference.
Clare O’Brien is an associate at Cobbetts