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Gene Therapy Can Help Improve Cystic Fibrosis LONDON, ENGLAND -- March 19, 1999 -- A study reported in this week’s issue of The Lancet shows that gene therapy can improve at least one of the underlying abnormalities in cystic fibrosis, a disease that causes chronic lung disease. This illness, one of the commonest genetic diseases, is caused by an abnormality in the gene CFTR. Professor Eric Alton and colleagues at the National Heart and Lung Institute and the Royal Brompton Hospital in London, England, and the Genzyme Corporation, Framingham, MA., administered a normal version of CFTR to eight patients with cystic fibrosis. There was significant correction of one of the characteristic biochemical abnormalities. The protein for which CFTR is the genetic code is called the cystic fibrosis transmembrane conductance regulator. One of its functions is to control transport of chloride ions across the epithelia (membranes) of organs such as the lungs and the digestive tract. When the protein is abnormal, as in cystic fibrosis, chloride movement is impaired. This disturbance, together with the increased sodium absorption that occurs in the disease, leads to excessive secretion in these organs and makes affected people susceptible to infection. Alton and colleagues combined the CFTR gene with a lipid and administered the mixture to patients in the form of an aerosol to the lungs (as is used in treatment of asthma, for example) and to the nose. Eight further patients received placebo (dummy) aerosols, without the gene mixture, so that the researchers could assess the efficacy of the active treatment. Administration of the gene to the lungs was followed by about 25 percent restoration of normal chloride transport in the patients who received the CFTR gene treatment but not in the placebo-treated patients. There was no effect on the other defect, sodium absorption. "Gene therapy for cystic fibrosis continues to make steady progress towards becoming a realistic therapeutic option for the disease," they write. Related Links: The Lancet. E-mail this page to a friend or colleague! To print, use this version