Navigation Links
Scientists develop first ever drug to treat 'Celtic gene' in cystic fibrosis sufferers

An international research team led by Queen's University have developed a ground breaking treatment for Cystic Fibrosis sufferers. The new drug will benefit sufferers who have the 'Celtic Gene', a genetic mutation which is particularly common in Ireland.

The study, which was carried out by scientists at Queen's University Belfast, the University of Ulster, the Belfast Health and Social Care Trust and teams of researchers in Europe, USA and Australia found significant improvement in lung function, quality of life and a reduction in disease flare ups for those receiving the new treatment.

The drug (VX-770) is a significant breakthrough not only for those with the 'Celtic Gene', known as G551D, but also for all other Cystic Fibrosis sufferers as it indicates that the basic defect in Cystic Fibrosis can be treated. This is the first drug aimed at the basic defect in Cystic Fibrosis to show an effect. It is still too early to determine whether this treatment will improve life expectancy but the improvements in the breathing tests and the reduction in flare-ups would suggest survival will be better.

Stuart Elborn, Centre Director in the Centre for Infection and Immunity at Queen's University and co-leader of the study said: "The development of this drug is significant because it is the first to show that treating the underlying cause of Cystic Fibrosis may have profound effects on the disease, even among people who have been living with it for decades. The remarkable reductions in sweat chloride observed in this study support the idea that VX-770 improves protein function thereby addressing the fundamental defect that leads to CF."

Dr Judy Bradley, from the University of Ulster said: "This drug opens the defective channel in the lung cells of people with Cystic Fibrosis and allows proper lung clearance of bacteria. This is a ground breaking treatment because it treats the basic defect caused by the gene mutation in patients. Correcting the cells with this mutation shows that treatments aimed at the basic mutation can work leading to improvements in lung function and symptoms."

Dr Damien Downey, from the Belfast Health and Social Care Trust said: "The success of this study illustrates the benefits that come from collaborative work here in Northern Ireland. Not only will this breakthrough help patients in Ireland and the UK but it has the potential to change the lives for those with Cystic Fibrosis around the world. As a result of the recent work researchers from Queen's University, University of Ulster and clinicians from Belfast Health and Social Care Trust have been selected to join the European Cystic Fibrosis Society Clinical Trials Network. This means Cystic Fibrosis researchers in Northern Ireland will be collaborating with their European counterparts to work toward improved treatments for Cystic Fibrosis on a global level. "

The new drug will be submitted for licensing in the Autumn of this year and is expected to be available to patients by as early next year.


Contact: Claire O'Callaghan
Queen's University Belfast

Related medicine news :

1. Scientists Turn Memory On, Off in Rats With Flip of Switch
2. Scientists Pinpoint Parts of Brain That Help People Recognize Themselves
3. Several methods for early diagnosis of Alzheimers disease developed by European scientists
4. Scientists image beginning stages of ovarian cancer growth with time-lapse technique
5. Scientists identify key component in lethal lung cancer complication
6. Swine flu spread was much wider than first thought, scientists say
7. Einstein scientists find crucial molecule involved in spread of breast cancer
8. Scientists create humanized mouse model for hepatitis C
9. Scientists unlock potential of frog skin to treat cancer
10. The New York Stem Cell Foundation awards fellowships to 7 innovative stem cell scientists
11. UCSB scientists discover new direction in Alzheimers research
Post Your Comments:
(Date:6/26/2016)... ... ... blind and certified personal trainer is helping to develop a weight loss fitness plan that ... the two major problems leading the fitness industry today:, , All ... They don’t eliminate all the reasons people quit their exercise program , ...
(Date:6/25/2016)... ... June 25, 2016 , ... ... and applications at AcademyHealth’s Annual Research Meeting June 26-28, 2016, at the Hynes ... important health care topics including advance care planning, healthcare costs and patient and ...
(Date:6/25/2016)... ... June 25, 2016 , ... The temporary closing of Bruton Memorial Library on June 21 due ... up a new, often overlooked aspect of head lice: the parasite’s ability to live away ... a common occurrence, but a necessary one in the event that lice have simply gotten ...
(Date:6/25/2016)... , ... June 25, 2016 , ... First Choice ... States, named Dr. Sesan Ogunleye, as the Medical Director of its new Mesquite-Samuell Farm ... Medical Director of our new Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical ...
(Date:6/25/2016)... , ... June 25, 2016 , ... ... and non-athletes recover from injury. Recently, he has implemented orthobiologic procedures as a ... area —Johnson is one of the first doctors to perform the treatment. Orthobiologics ...
Breaking Medicine News(10 mins):
(Date:6/26/2016)... , June 26, 2016 ... care operating models within the health care industry is ... financial efficiency , Deloitte offers a suite of ... business issues impacting efficient cost optimization: labor resource analysis, ... These services facilitate better outcomes and better economics ...
(Date:6/24/2016)... Dublin ... of the " Global Markets for Spectroscopy Equipment" ... This report focuses on the global ... including its applications in various applications. The report deals ... three main industries: pharmaceutical and biotechnology, food and beverage, ...
(Date:6/24/2016)... June 24, 2016 Research and Markets ... for Companion Diagnostic Tests" report to their offering. ... Companion Diagnostics The World Market for Companion ... medicine diagnostics. Market analysis in the report includes the following: ... (In Vitro Diagnostic Kits) by Region (N. America, EU, ROW), ...
Breaking Medicine Technology: