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New drug cocktail offers hope to those with cystic fibrosis

By Victoria O'Hara

Research led by a Belfast scientist has discovered a potentially groundbreaking treatment for cystic fibrosis sufferers that could help transform the lives of patients round the world, a leading charity has said.

The Cystic Fibrosis Trust said the findings of a major trial jointly spearheaded by Professor Stuart Elborn from Queen's University in Belfast "open up a new front" in the fight against the disease.

The trial on 1,108 patients, reported in the New England Journal of Medicine, showed a combination of drugs could bypass the genetic errors that cause the disease and may increase life expectancy.

Cystic fibrosis affects around 10,000 people in the UK and is a life-shortening genetic condition. Just half of sufferers live to celebrate their 40th birthday.

Prof Elborn, who led the European section of the trial and is regarded an international authority in respiratory medicine, said: "These treatments are potentially transformative for young people.

"They still die in their 20s and 30s. Having a potentially disease-modifying therapy is critically important.

"It is not a cure, but it is as remarkable and effective a drug as I have seen in my lifetime."

The faulty gene is carried by over two million people in the UK, most of whom have no idea. If two carriers have children, there's a one-in-four chance their child will have the condition. Cystic fibrosis affects the internal organs, especially the lungs and digestive system, by clogging them with thick, sticky mucus. This makes it hard to breathe and digest food.

Around 250 adults are currently inpatients at the cystic fibrosis unit at Belfast City Hospital.

Janet Allen, director of research and care at the Cystic Fibrosis Trust, said: "These results open up a new front in the fight against cystic fibrosis and this combination therapy looks set to be an important additional treatment option that could improve the lives of many.

"This therapy is currently being examined by European regulators for use across the UK. If approved, it will fall to the NHS to fund what is expected to be a high-cost drug.

"As this leading edge of science continues to be explored and better understood, we are hopeful that a future of personalised medicines is increasingly within reach."

Case study: Lorraine Mullan (32) from Poleglass in west Belfast is studying accountancy and is a full-time mum-of-two.  She has cystic fibrosis but says such scientific news of possible drug breakthroughs helps to give hope.

"The daily routine can be tough. I have two children so it is just fighting to get everything in. When I get up in the morning I have to take two nebulisers first thing, I have to take tablets before breakfast, tablets before lunch, tablets before dinner. I have another three inhalers and two nebulisers to take before bedtime. That is on a good day.

“On a bad day I would have to take more nebulisers, just to keep my chest clear. I do physiotherapy myself around two or three times a day. If I’m feeling unwell that can go up to five times. My two boys Finn (2) and baby Lucas (11 months) are carriers of the gene.

“Prof Elborn is still a consultant in the City Hospital and concentrates on research, but to me there is nobody on the planet like him.

“He is just amazing. I go to the City twice a month when I am well, when I’m not it would be every fortnight.

“My immune system is so low and it is very easy to pick something up, so you have to go in for IV antibiotics and intensive physio. As I get older — I’ll be 33 this year — I do notice the difference. I would have more hospital admissions than a few years ago. I would consider myself to have quite mild CF and controlled, but I’ve lost a lot of friends over the years. Some were on the transplant list, but I feel very lucky that mine is controlled quite well. But I have an amazing family,  I couldn’t do without them. My partner John is fantastic and very hands-on supporting me. We are getting married in October and just getting the RSVPS out, so we are really looking forward to that.

“You are just waiting for somebody coming up with something. It can bring you hope, but it can be frustrating. Prof Elborn has put all this hard work in getting this drug and trial — but it still has to be approved for funding. It always comes down to money.

“When you are the person suffering with it you don’t think there should be a price tag on life."

Belfast Telegraph


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