The Future for Gene Therapy and Treating Cystic Fibrosis Patients


Cystic fibrosis is caused by a mutation in the gene CFTR. This gene involves a protein that affects the chloride on cells; without this chloride the surface of the cells do not attract as much water and the mucus around the cells becomes sticky and thick.

Although the disease also affects the liver, pancreas, and bowels, the thick and sticky mucus in the lungs is often the most deadly factor in the disease. This mucus can clog the airways and make patients more likely to contract deadly lung infections, lung disease, and inflammation.

Even though the cystic fibrosis causing gene was found back in 1989, a gene therapy for the disorder has yet to be developed. There are over 2,000 different mutations in over six classes for this gene, which makes treatment very difficult. After the cystic fibrosis gene was discovered, many companies began the search for a successful gene therapy, but many have since given up and there have been no therapies created that can be used for all cystic fibrosis patients.

Deborah Gill, a British professor at the University of Oxford who is working on a gene therapy, spoke about the benefits of a successful one. In 2015, the University completed one clinical trial, but the benefits were so moderate that researchers are currently working to modify the therapy to increase its effectiveness. The issue with medicines so far has been delivering the therapy to the lungs. They believe that this could be completed in 3-5 years. If the trial is successful, it would remove the need for any other medications for lung function for cystic fibrosis patients.

It is important to note that these new therapies only focus on the effect that the disease has on the lungs. The complications in the lungs are the most severe of the symptoms and lung disease is usually the cause of fatality in cystic fibrosis patients, but scientists are beginning to look at other issues with the disease such as the bowels, pancreas, and liver.

For future endeavors, Verona Pharma in the UK is working on a new medicine that acts to open the bronchioles and also as an anti-inflammatory for cystic fibrosis patients. They have already had positive results from a phase 2 trial.

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