UK Gene Therapy Trial For CF "A Success"

Speaking at a British Association conference in Loughborough, UK, Duncan Geddes of the Royal Brompton Hospital in London said that a small double-blind gene therapy trial of 15 patients with cystic fibrosis had produced a 20% correction of the gene defect in nasal cells in patients.

The UK trial utilized a liposome delivery system containing genes coding for the cystic fibrosis transmembrane regulating (CFTR) factor which was administered to the cells of the nose. CFTR controls the flow of chloride ions across cell membranes and its functioning is impaired in CF.

A US trial conducted at the National Heart, Lung and Blood Institute using an adenovirus as the administration vector for the additional genes was discontinued after one of the recipients, who received a high dose of adenoviral vector, developed a fever and inflammation of the lungs. The trial was restarted with a lower dose vector and is currently ongoing. Dr Geddes said that the liposome vector appears to be safer than viral administration as there is no risk of an immune response to the vector. "We had no problems and the results are encouraging," he added.