An evening of unproved treatmentBMJ 1996; 312 doi: https://doi.org/10.1136/bmj.312.7027.385 (Published 10 February 1996) Cite this as: BMJ 1996;312:385
- Douglas Carnall
Two documentaries, both broadcast on the same evening, explored two very different approaches to conceptualising and coping with disease. Horizon followed two teams of scientists competing to be the first successfully to reinstall a normal gene in place of the faulty gene of cystic fibrosis, while Cutting Edge brought us Cari Loder, who attributed her remission from her disabling multiple sclerosis to a cocktail of antidepressants, amino acid supplements, and B12 injections.
Shored up in her belief in her treatment by the four occasions on which, having omitted the morning dose of one of the ingredients, she had been unable to climb the stairs, Ms Loder published her findings in Laboratory News. Her phone has not stopped ringing since. Those who rang were initially entered in a trial of the treatment, the success of which was assessed by the buoyant Ms Loder over the phone. Early excitement at the “97.5% success rate” in the first 80 patients had calmed to a somewhat more modest six patients in 10 by the time she was filmed discussing the design and conduct of a randomised trial to answer the “cynicism, scepticism, and vitriolic attacks from neurologists and the MS charities.” Her own relapse, despite treatment, was attributed to stress rather than treatment failure.
Ms Loder purveyed hope in great quantity: the scientists searching for an effective method of inserting healthy genes into the bodies of patients with cystic fibrosis offered some, but not much hope. The funeral of Sean Conroy, who died aged 21 from respiratory complications of cystic fibrosis, illustrated for Horizon's viewers how much the gene therapists still have to achieve. Sean had been one of 15 participants in a British trial designed to test the ability of liposomes to transport healthy DNA into the cells of his nasal mucosa, an approach that would eventually be described as “a safe procedure that doesn't work very well.” A rival team, headed by Professor Ron Crystal, fearlessly doctored some adenovirus with the healthy gene and introduced it into the lungs of a patient with cystic fibrosis, emerging from the bronchoscopy suite to all-American applause. The applause was premature: the gene had been taken up by some cells, but in too small amounts to have any significant effect on the disease.
The race is still under way. If money will buy the answers the Americans seem likely to win. The cramped and dilapidated surroundings of the British team made telling contrast with the vast open spaces of the American facilities. It will be a race worth winning—the cool headed calculation of the biotechnology firm that is investing hard in the research is that successful gene therapy for cystic fibrosis will be worth more than $500 million. Cari Loder's treatment costs pounds sterling15 a month, and she is about to publish her life story. The patent for her treatment is lodged in 77 countries, and if the trial bears fruit she may also reap the rewards. I believed her though, when she said that money was not her prime concern. What drives her is anger at a medical establishment “whose eyes glaze with condescension” at the prospect of a non-medic thinking she has found the cure “when they have failed.”
Standing the programmes side by side made a whole bigger than the sum of its parts. The scientists were rather undemonstrative and serious people who spent a lot of time in dingy rooms doing interesting things with micropipettes. Ms Loder, a free spirit in the park with a mobile phone, was warm, defiant, protective, and optimistic. The success of Ms Loder's treatment may indicate how many doctors out there fail to diagnose and treat depression among patients with multiple sclerosis, but both programmes showed us that all healers can be impotent in their attempts to reverse the course of the disease. The danger of the illusion of the rational cure is that in trying to separate out the power of positive thinking from the effects of treatment, we throw away the power of positive thinking as well.—DOUGLAS CARNALL, BMJ