THE parents of three-year old twin boys born with a debilitating disease have pleaded for medicine to be put before profit.

Victoria and Ian Blair’s two sons James and Alex both have cystic fibrosis (CF), a cruel genetic illness that shortens and reduces quality of life.

New precision medicines exist to target the root cause of the disease, but a stalemate over the costs between National Health England and US drug developer Vertex Pharmaceuticals has prevented them getting hold of the potentially lifesaving treatment.

And now the Blairs, who live in Hewelsfield with their twin sons and older daughter Sophie, have joined other families to ask Vertex to put the lives of patients before financial gain.

They have backed a letter to Dr Jeffrey Leiden, CEO of Vertex, urging him to prioritise global availability of the medicines, particularly a drug called Orkambi, which costs £104,000 per patient per year in the UK.

Mum Victoria said that when the twins were first diagnosed, she “felt as though an articulated lorry had just smashed straight into me.”

“Both of our sons, James and Alex, have had multiple health issues throughout their young lives. James particularly had struggled from birth as he had a tracheal oesophagal fistula (TOF) and a perforated bowel,” she explained.

“He had to have two operations, was dosed up on morphine and had tubes and wires everywhere. He looked a very sorry sight.”

After the diagnosis of cystic fibrosis, both boys were hospitalised with bronchiolitis.

“James had only been out of hospital for six days at that point,” added Victoria. “In the past three years, we have grown used to the daily tasks that CF brings – the boys have a strict treatment and physiotherapy regime that needs to be undertaken daily.

“But I do resent not only how often it makes our boys feel so poorly (usually at night), but also how much it steals me away from my older daughter Sophie, who is so desperate for my attention.

“I am both optimistic and pessimistic about what the future holds. Pessimistic about the health of my boys, the effect on my daughter, the repeated hospitalisations and trying to avoid germs and bugs.

“I am optimistic about the precision medicines that are either licensed already or in development. A triple therapy that shows great promise and can treat 90 per cent of those with CF is currently in trials.

“But now that negotiations between Vertex and NHS England have reached a stalemate, it is very hard to comprehend that although patients in other countries are benefitting from these amazing drugs, we still do not have access.

“Every day that these negotiations are at deadlock, CF continues to wreak havoc on my sons’ health and quality of life.”

Cystic fibrosis causes a build-up of thick, sticky mucus in the lungs and other organs, which can lead to chronic lung infections and progressive lung and digestive system damage.

In the letter sent to Vertex, campaigners say: “It is a truly amazing achievement that your drugs can one day end the threat to life that has hung like a dark cloud around the world for decades.

“However, Vertex’s inspirational science and dedication must now be matched by a determination to rid the globe of the scourge of early death from CF.

“At the recent JP Morgan Healthcare Conference you stated that only 18,000 of the 37,000 patients eligible for your three licensed drugs are being treated.

“We are asking for immediate treatment with current drugs for all patients and to have the prospect of even better therapies in the future. We represent patients who remain untreated all around the world – over half.”

Although some countries have obtained the drug – including the Republic of Ireland and Australia - many other countries, including England and Wales, are still denied access, although Scotland has an interim deal in place.

More than 220 people who would have been eligible for the drug in the UK have died over the last three years, claim campaigners.

Last year, a petition that received more than 100,000 signatures in 10 days led to a government debate and widespread publicity but, despite assurances from Prime Minister Theresa May, the situation has now reached deadlock.

In response to the letter, Dr Leiden said: “Vertex shares the responsibility with governments and regulators to do all that we can to agree to a fair price that reflects the value of these medicines in all countries where they are needed.

“This in turn will allow us to keep investing in the possibility of a world without CF and continuing our important work in other serious diseases.

“And importantly, in the countries where access is still not agreed, our position is always the same – we are doing everything we can to work constructively with the government authorities to find workable solutions.”

The letter to Dr Leiden can be viewed at www.cfsupportgroup.org/letter-to-jeffrey-leidenThe petition can be found at: www.petition.parliament.uk/petitions/231602