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Gene Therapy Breakthrough for Cystic Fibrosis

Gene Therapy Breakthrough for Cystic Fibrosis

Cystic fibrosis is one of those conditions that we are yet to have found a cure for. All we can hope for at the moment is to develop treatments that help elongate the sufferer’s life and make it more worth living.

And the latest trial, published in The Lancet Respiratory Medicine, might just be what we have been waiting for.

The condition, often shortened to CF, is caused by a faulty gene and affects the lungs and digestive system, by clogging them with thick mucus. Symptoms of CF include a persistent cough, difficulty in gaining weight, recurring chest and lung infections, and unusually salty sweat.

In the UK, it is believed that one in every 2,500 babies are born with CF. Patients are often diagnosed in early childhood, and while they usually live into adulthood, the average lifespan for someone with the condition is only about 41.

As we said, there isn’t a cure just yet, only treatments aimed at easing the symptoms, or at least making the condition easier to live with.

Now, about that trial we mentioned!

The small study, led by Professor Eric Alton from the Imperial College London, involved 136 CF sufferers over the age of 12. The participants stopped using their regular medication and replaced it with an inhaled solution that was given once a month over the course of a year.

Half of the group was randomly assigned a saline solution placebo. The remaining study participants were given a virus to inhale. This virus contained healthy copies of the gene that causes cystic fibrosis, which had been altered using gene therapy.

All of the solutions were inhaled via a nebuliser, which the patients would be accustomed to because of their condition. This device produces a fine spray of liquid that is inhaled straight into the cells in the lungs, where it is needed.

The lungs of the patients receiving just the placebo showed an average decline of 3-4% over that year-long trial. However, the patients who received the genetically-altered healthy cells didn’t really show any decline, and even those with the most clogged lungs at the beginning of the study showed an improvement of around 3%.

The national team behind the trial haven’t yet found a way of making a more permanent change to the faulty gene that causes CF, and the effect is only modest and depends on the patient. But the team believes that with more doses per month, and combined with their regular treatment, the results could be vastly improved. Though, this can only be shown in more trials, and on a much larger scale.

Professor Alton explained that the lungs are possibly one of the worst organs for the condition to attack because of how well they are defended. Usually in gene therapy, for example, bone marrow is taken from the patient and the work is carried out under controlled lab conditions and put back into the patient.

“That is the low-hanging fruit,” the professor explained. “We have gone for high-hanging fruit, but I am not at all sorry we have.”

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, said the benefit of using gene therapy to help CF patients is that it could reduce the need for a daily cocktail of medication.

“We will, therefore, continue to invest in innovative genetic research… to develop advancements,” he concluded, “which will make further progress towards our goal of a life unlimited by cystic fibrosis.”

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