Gene Therapy Helps Cystic Fibrosis Sufferers - Study
08:37 a.m. Mar 19, 1999 Eastern

By Patricia Reaney

LONDON (Reuters) - Gene therapy can improve a major lung defect in patients suffering from cystic fibrosis, British researchers said Friday.

Cystic fibrosis is a chronic lung disease that causes a buildup of fluid in the bronchial tubes, making the lungs vulnerable to infection. The incurable illness results from an abnormality in the CFTR gene.

In one of the first controlled medical trials using gene therapy in lungs, doctors at the Royal Brompton Hospital in London and Genzyme Corporation in Framingham, Massachusetts, had patients inhale a normal version of the CFTR gene.

``It is the first time a large amount of a gene has been inhaled,'' Duncan Geddes of the Royal Brompton Hospital said in a telephone interview.

The inhaled gene therapy restored some of the movement of chloride in patients' lungs. Chloride movement and sodium absorption are the fundamental biological abnormalities of cystic fibrosis patients which makes them more susceptible to infection.

Geddes and his colleagues, whose research is published in The Lancet medical journal, gave eight patients a single dose of the gene therapy through an aerosol inhaled through the mouth or nose. Eight other patients had a placebo, or dummy treatment.

Only one dose of the therapy was allowed for the trial.

The gene therapy restored chloride movement by 25 percent in patients for about 10 days. It had no effect on sodium absorption.

``We probably would have to get much higher levels of correction (in the chloride) to get that to improve as well. If we got complete correction of the chloride movement we might well have seen correction of the sodium,'' he said.

``The belief is that the abnormality in the chloride channel leads to an abnormality in sodium. Correct one and you would expect to correct the other.''

Although a 25 percent improvement in chloride movement is encouraging and shows that the treatment is working, Geddes believes he and his colleagues can do much better.

Patients who received the gene therapy had mild side effects such as a temperature and headache, but the researchers said those can be alleviated as the treatment is improved.

``We are finding ways of improving the efficiency of transferring the gene in and modifying the chemical nature of the gene so it doesn't produce the side effects,'' Geddes said.

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