GENE-THERAPY FOR CYSTIC-FIBROSIS IN HUMANS BY LIPOSOME-MEDIATED DNA TRANSFER - THE PRODUCTION OF RESOURCES AND THE REGULATORY PROCESS

The number of clinical trials using gene transfer technology, either a ctive or under discussion, is increasing rapidly. However, little info rmation is available describing the regulatory procedures or safety sp ecifications that must be considered before initiation of such trials in Europe. We describe the procedure used by our group to produce reso urces for the first stage of a phase I trial of liposome-mediated gene therapy for cystic fibrosis. The current lack of written and co-ordin ated guidance from the numerous interested regulatory agencies within the UK and Europe makes determination of the appropriate safety specif ications and procedures for these novel trials difficult, as does the fact that some new agencies (such as the Genetic Therapy Advisory Comm ittee in the UK) and some which are unfamiliar with clinical tests (su ch as the Department of the Environment) are involved as well as the M edicines Control Agency. In addition, we estimate that the realistic c ost of these trials, which in many cases will have to be covered from research budgets provided by government agencies or medical charities, could lead to delays in the clinical application of this important ne w therapeutic strategy.