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Phase Two Gene Therapy Clinical Study For Cystic Fibrosis Begins

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Phase Two Gene Therapy Clinical Study For Cystic Fibrosis Begins

This study that will hopefully prove that gene therapy developed by the UK Cystic Fibrosis Gene Therapy Consortium can improve the health of patients suffering from cystic fibrosis has begun. Phase two clinical study begins this month, led by researchers from Oxford University, Imperial College London and the University of Edinburgh. It will be based on the observation of 130 patients that will breathe through an inhaler the working copy of the cystic fibrosis gene once a month for a year.



Dr. Deborah Gill of the Nuffield Department of Clinical Laboratory Sciences at Oxford University said: “It's a worldwide first in terms of the length of the study, the number of patients involved and the number of doses of gene therapy. By giving the therapy over a whole year, we will have the best chance yet of seeing an improvement in patients.”

Around 9,500 people in the UK are affected by cystic fibrosis, a disease caused by a faulty gene. Until now the conventional treatment for this disease focused only on the symptoms. By alleviating the symptoms, the  treatment extended the life expectancy of the patients which is now around the age of 35 years. This disease affects mucus glands in the lungs and digestive systems, leading to thick, sticky mucus.

This new therapy should be more effective as it focuses on the basic defect, declared Professor Eric Alton, the coordinator for the consortium from Imperial College London. He adds: “This trial will assess if giving gene therapy repeatedly for a year will lead to the patients’ lungs getting better. Eventually we hope gene therapy will push cystic fibrosis patients towards a normal life expectancy and improve their quality of life significantly.”

The treatment implies that patients inhale a gene through a nebulizer. This method was proved efficient in a previous clinical trial which showed that the working copy of the cystic fibrosis gene enters the lung cells and that it continues to work for a period of weeks and months. This new clinical trial tries to answer the question whether the gene therapy reduce the amount of mucus, inflammation and infection and therefore improve the lung function.

Additional laboratory studies will be made to develop a more efficient delivery method for the gene therapy involving modifies viruses that are able to carry the working cystic fibrosis gene directly into the cells: “The new virus delivery approach has never been tried before but it could be more efficient. It is specifically designed to deliver the gene therapy to the lungs, but it will take several more years of development before it gets to the point of human trials,” said Dr. Gill.

The research will take place with the £3 million funding from Medical Research Council (MRC) and the National Institute for Health Research (NIHR) after facing uncertain future due to lack of funding after being supported by the Cystic Fibrosis Trust for over a decade.

Professor Eric Alton of Imperial College London said: “With funding for this key part of the consortium’s program secure, we will begin preparations for the trial immediately.”

The results of the trial will be published in 2014, but until then readers can find regular progress on the UK Cystic Fibrosis Gene Therapy Consortium’s website.