Families support cystic fibrosis gene therapy trials in children

30 May 2006

Cystic fibrosis (CF) is a childhood- onset life-limiting illness (currently, average life expectancy is less than 25 years, although this figure is rising as treatments improve) associated with high morbidity (ill-health) that affects the respiratory and digestive systems. CF is caused by defects in the CFTR gene on chromosome 7; because the condition is a single-gene (Mendelian) disorder, it has the potential to be treated by gene therapy, provision of functional copies of the CFTR gene into the bodies of affected individuals. The actual and potential risks associated with any form of gene therapy generally require that clinical trials must be performed initially inadults.

However, a new report in the Journal of Medical Ethics questions whether it is in fact unethical to deny children access to participation in appropriate clinical trials from which they might derive clinical benefit. Indeed, the benefit might be substantially greater (and scientifically easier to demonstrate) in children, as gene therapy could prevent the onset of significant lung disease. Lead researcher Dr Adam Jaff

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