Gene therapy in the clinic: whose risks?
Current experience with gene therapy for X-linked severe combined immunodeficiency disorder (X-SCID) suggests that we might now be at the point where it can be considered the 'standard of care'. This therapy carries an unquantified risk of leukaemia, raising important questions for regulators. How dangerous does a potentially curative therapy for a fatal illness need to be before we call it unsafe. How uncertain does a risk need to be for us to regard a therapy as still 'experimental'? Whose interests should prevail? Here I argue that in X-SCID it is the parents and children whom we should listen to first and foremost. How far does this patient-centred approach to licensing therapies extend? Can it be given a rational basis?
Ashcroft
Richard E
RE
Imperial College London, Medical Ethics Unit, 324 Reynolds Building, St Dunstan's Road, London, W6 8RP, UK. r.ashcroft@imperial.ac.uk
D003257:Consumer Product Safety / Q000331:legislation & jurisprudence*; D015316:Genetic Therapy / Q000009:adverse effects / Q000941:ethics* / Q000331:legislation & jurisprudence*; D033161:Government Regulation; D006801:Humans; D007938:Leukemia / Q000209:etiology / Q000235:genetics / Q000517:prevention & control; D010818:Practice Patterns, Physicians' / Q000941:ethics* / Q000331:legislation & jurisprudence*; D011639:Public Opinion; D011640:Public Policy; D018570:Risk Assessment / Q000941:ethics* / Q000331:legislation & jurisprudence; D012307:Risk Factors; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D014481:United States