Research finds majority of people with cystic fibrosis can be treated successfully with three-drug combo

Research finds majority of people with cystic fibrosis can be treated successfully with three-drug combo
St Vincent's Hospital Dublin

Groundbreaking research has found that the majority of people with cystic fibrosis can be treated successfully.

Researchers at Queen’s University in Belfast and at the Cystic Fibrosis Unit at St Vincent’s Hospital Dublin, have identified how a combination of three drugs, known as triple therapy, can tackle the underlying cause of the incurable disease.

The drug, Trikaftatm, targets the root cause of cystic fibrosis (CF), a genetic condition that clogs up the lungs and digestive system, making breathing difficult and often resulting in an early death for those affected.

CF is an inherited chronic disease that primarily affects the lungs and digestive system of about 1,300 children and adults in Ireland and 70,000 worldwide.

That figure, according to Philip Watt of Cystic Fibrosis Ireland, is set to increase by 75% in adults and 25% in children in this country according to ongoing studies by 2025.

“This new drug Trikaftatm will benefit 90% of sufferers worldwide, lung function is expected to increase dramatically and will result in a 60% decrease in hospitalisation. This is a hugely welcome advancement. Many of the trials by the Queen’s University researchers were carried out at several centres in Ireland including St Vincent’s Hospital Dublin.

“Dr Francis Collins, an American researcher who discovered the CF gene in 1989 has already said this is the breakthrough in research he has been waiting for. Many of the trials prior to this did not achieve as much as this study has.

“Dr Collins who is the director of the National Institutes of Health writing in The New England Journal of Medicine and the highly respected Lancet medical magazine over the weekend, said these findings indicate that it may soon be possible to offer safe and effective molecularly targeted therapies to 90% of persons with cystic fibrosis.

“He said that this should be a cause for major celebration and the ‘best day ever’ for all of us traveling down this long road together will be the day when the more than 70,000 persons with cystic fibrosis worldwide do not need to take drug therapy at all and there finally is a permanent cure for cystic fibrosis that works for everyone.”

Mr Watt added: “The Food and Drugs Administration in the US has approved this drug five months ahead of time and now the European Medicines Agency is reviewing it. As a result we would expect this drug to be on the market here by the Summer of next year to those over 12-years-of-age. We would be hopeful that will include children aged as young as two in the future.

“The numbers of adults and children increasing in Western Europe is down to advancements in related drugs and quality of services.

Talks and negotiations with the Health Service Executive on this new announcement will, I’m sure, commence as a pipeline deal on future drug advancements was agreed with them in 2017 when Orkambi was finally approved. So far we are not privy to the potential costs of this new drug.

However, it is estimated that the drug may cost around $311,000 or €279,000 annually by Vertex, a pharmaceutical company that produces other CF drugs.

Ireland has the highest incidence of CF in the world with one in 19 Irish people being said to carry one copy of the altered gene, with three times the rate of the United States and the rest of the EU.

The organisation has pointed out that it is important to have a network of centres of expertise in dealing with disease.

A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections; and obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.

Researchers believe it is possible to help control the symptoms and delay complications to make the condition easier to live with.

Dr Damian Downey, is a Clinical Senior Lecturer at the Wellcome-Wolfson Institute for Experimental Medicine, Queen’s University Belfast and co-author on the worldwide trial, set-up to assess the safety and efficacy of a new triple-drug combination called Trikaftatm in patients with CF aged 12 years and older.

The study involved a four-week, randomised, active-controlled trial in 107 patients who had two copies of the (F508del) most common gene mutation.

Dr Downey said: “The trial was a success in demonstrating that this drug combination can potentially treat up to 90% of people with CF by addressing the underlying cause of their disease.

“This new triple therapy has the potential to transform the lives of people with CF. It results in a significant improvement in lung function and quality of life and also reduces the frequency of chest infections. This treatment will likely alter the future of CF care.”

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