Call to allow gene therapy trials for children with cystic fibrosis

A paper has been published in the Journal of Medical Ethics that investigates the possibility of trialling gene therapy for cystic fibrosis (CF) in children with the condition. Conventional wisdom has generally suggested that it would be unethical to use untested new treatments, such as gene therapy, on children affected by a particular disease. But the new study describes how, in the specific case of CF, children with the disease may find a unique benefit to the treatment not available to affected adults, due to the progressive nature of the lung damage that occurs in the disorder. The researchers also used questionnaires to investigate parental attitudes toward enrolling their own children into gene therapy trials.

CF is a life-limiting illness which causes ongoing problems in affected individuals from an early age. A faulty gene on chromosome seven results in defective production of a protein that regulates cellular ion transport. It is thought to be this disruption to ion transport that results in the characteristic sticky mucus, which blocks airways and leads to lung damage and eventually death. The disease also affects the digestive system and CF patients experience increased bacterial infections. People with CF born in the 1990s are now predicted to survive up to around age 40, however in 2003 the median age of death of patients was 24.2 years. As CF is a single gene defect, it is a suitable candidate for gene therapy, where an attempt would be made to insert a non-defective copy of the CF gene into deficient cells.

In the paper the team, based at Great Ormond Street Hospital in London, asserts that not only may it be unethical to deny children access to a therapy that may provide clinical benefit to them, but that furthermore because children do not yet suffer from the lung damage characteristic of patients later in life, the treatment may be more effective for them. Talking to the BBC about the paper, lead researcher Dr Adam Jaffe said 'we could be denying children life enhancing treatment on inadequate grounds. There are scientific and medical reasons why the treatment might work better in children. The main aim is to halt the decline in lung damage, which begins soon after birth so it makes sense to start treatment in early childhood'.

The researchers present results from a questionnaire given to parents of affected children aged between six months and 17 years being treated in their unit. The response rate was 48.8 per cent and represented families with a total of 80 children. Parents have ethical and legal rights to make decisions to optimise the care for their child. In the survey 91 per cent said that they would consider enrolling their child into a gene therapy trial, with 99 per cent believing that it was ethically sound for children to be given the opportunity to take part in gene therapy clinical trials, provided that they were carefully run, with safety issues as the priority.