New drug trialled by Queen’s brings hope for cystic fibrosis patients 
18 May 2015
Clinical investigators from the School of Medicine, Dentistry and Biomedical Sciences at Queen’s having been taking part in an international project which is bringing fresh hope to those with cystic fibrosis (CF).
The team carried out trials in centres across the world with over 1,100 people who have the most common form of cystic fibrosis, F508del, a life limiting genetic disease which can affect the lungs, liver, pancreas and kidneys.
The researchers found that Orkambi – a combination of two drugs Lumafactor and Ivacaftor – can improve lung function and reduce hospital treatments for cystic fibrosis sufferers. Orkambi is the first therapy that targets the underlying cause of CF, in about 45% of cases.
The results of the trial also showed a reduction in the number of hospital courses of antibiotic treatment, an improvement in patient’s breathing tests, weight and quality of life.
Co-author of the research, Professor Stuart Elborn (pictured above), Dean of the School of Medicine, Dentistry and Biomedical Sciences at Queen’s, said: “These results represent a further major advance in finding treatments which correct the basic problem in cystic fibrosis and improve the lives of patients living with the condition.
“This is the latest example of the commitment of researchers and staff at Queen’s and the Belfast Health and Social Care Trust to advancing knowledge and achieving excellence for the benefit of everyone in society.”
The combination treatment was developed by Vertex, a pharmaceutical company based in Boston MA, and is now undergoing assessment for approval and clinical use. The US Food and Drug Administration is expected to make a decision on the approval of Orkambi by early July. An application for its approval has also been sent to the European Medicines Agency, which means that the drug could be available in the UK/Ireland as early as next year.
Cystic fibrosis is a genetically inherited disease which affects the lungs and digestive system. CF produces a sticky mucus, which clogs the body’s organs and gives rise to infection, mainly in the lungs and pancreas.
The international research team that carried out the study involved scientists from the UK, Ireland, the United States, Australia, Italy, France and Canada.
For more on the cystic fibrosis research being carried out at Queen’s click here, or if you would like to support this medical activity, please contact Helen Surgenor, telephone: +44 (0)28 9097 3159.
Media inquiries to Andrew Kennedy, Queen’s Communications Office, on 028 9097 5384.
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