Stem cell research targets Cystic Fibrosis

Stem cell research targets Cystic Fibrosis

Following a substantial grant by the Evelyn Trust to Dr Ludovic Vallier and his talented team of researchers at the University of Cambridge, effective treatment for Cystic Fibrosis has moved a step closer.
Image of lungbud

Lung bud

Cystic Fibrosis is the most common life-shortening genetic disease that affects around 10,000 individuals and families in the UK today and many more thousands around the world. There is no cure and, although management of the condition has improved in recent decades, the disease still kills many people each year – statistics shows that only around half of people with Cystic Fibrosis will live past their forties.

Cystic fibrosis is caused by a faulty gene. This gene controls the movement of salt and water in and out of cells, so the lungs and digestive systems of patients become clogged with mucus, making it hard to breathe and to digest food. The disease is typically managed with physiotherapy, exercise, correct nutrition and treatments to improve lung function and control infections. More than 2.5 million people in the UK carry the faulty gene - around one in 25 of the population – and although most will not suffer from the disease, they can pass the gene to their children who may then develop the condition.

The development of an effective drug therapy for Cystic Fibrosis had been held back by the lack of modelling of the disease ‘in vitro’ – ‘test tube’ modelling, in laymen’s terms. Dr Vallier’s team in the Department of Surgery has developed a new approach to modelling Cystic Fibrosis in the laboratory using human induced pluripotent stem cells (hIPSC). These are stem cells generated by reprogramming cells from patients’ skin which can then be differentiated into almost any cell type.

The team’s first step was to develop a culture system that allowed the production of a homogeneous population of lung cells from hIPSC. Then stem cells were produced from cells taken frompatients with Cystic Fibrosis and reprogrammed as lung cells, allowing the condition to be modelled ‘in vitro’. Once that had been achieved, the team were able to test various drug therapies on the model and found that one in particular was very effective. This is a drug currently in atrial phase and the team hope that the results of their work will heighten interest in this therapy and accelerate investment in the trials.

“When most people think of the potential benefits of stem cells, they think of their use for therapeutic applications – regenerating damaged tissues or organs, for example. While these types of applications are certainly exciting, it is a long journey from lab to clinic. It’s the use of stem cells for the development of new drugs which will we believe will have the most immediate impact on human health,” explains Dr Vallier. “We are very hopeful now that our ‘in vitro’ modelling of Cystic Fibrosis will enable a real breakthrough in therapeutics for this disease that has been labelled ‘incurable’ for so long.”

The Evelyn Trust is enthusiastic in its support of ground-breaking medical research and has been proud to fund Dr Vallier and his team of world class researchers as they have pursued this very successful project over the last two years.

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