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My little boy’s life is a misery due to cystic fibrosis... the NHS can help by providing a vital new drug

By Victoria Leonard

The mother of a 15-month-old Downpatrick boy with cystic fibrosis is urging the Prime Minister to step in to provide a "life-changing" new drug on the NHS.

Jen Banks (33) said her son Lorcan Maguire is already showing signs of deterioration due to the genetic disease.

It affects around one in 19 people in Ireland and causes a build-up of thick, sticky mucus in the lungs, digestive system and other organs.

Campaigners are calling for the drug Orkambi, which is already available to cystic fibrosis sufferers in the Republic, to be introduced in Northern Ireland and the rest of the UK.

Last month, hundreds of letters were delivered to Theresa May from families and children asking for Orkambi. A protest calling for access to the drug will take place at Belfast City Hall on June 22 from noon until 2pm.

Orkambi is currently not available on the NHS in the UK, with the National Institute for Health and Care Excellence (NICE) saying that "for the benefits it offers, the cost of Orkambi is currently too high".

Ms Banks said she and husband Dermot knew nothing about cystic fibrosis when Lorcan was born.

"He is a gorgeous, wonderful child, but he is already showing signs of deterioration, with thickening of mucus in his lungs and inability to absorb nutrients from food.

"He has a cough all the time, and every day he has to take two nebuliser drugs, 20 enzymes, two vitamin tablets and two antibiotic tablets - and he is pretty well at the minute. He has to have physio two or three times a day when he is well, to get the mucus to loosen up.

"The main issue which causes death is what happens in the lungs with the mucus - they become a perfect breeding ground for bacteria, so they get infection after infection, then deteriorate.

"Every time we go out and someone coughs you panic, if you smell smoke you panic. We use antibacterial gel constantly.

"My father has Pseudomonas in his lungs and he and Lorcan have to stay three metres apart, which is heartbreaking.

"When he was born we were told by the midwife that life expectancy was up to 31, but these new drugs would mean he could live up until the age of around 47."

Ms Banks said that Orkambi represents a revolutionary treatment for cystic fibrosis.

She continued: "Other drugs only deal with the symptoms, whereas Orkambi actually helps the movement of salt and water through the cells, working at the root of the problem.

"In the Republic, they also have a pipeline deal whereby new drugs for cystic fibrosis are made available when they are developed, and the new drugs being developed now could help up to 90% of sufferers.

"They already have this drug in the Republic, America and across Europe, where it can be used from six years old."

In the absence of a Health Minister, Ms Banks is calling on Mrs May to take action. She added: "Lorcan will die from this unless something is made available. It's really heartbreaking for me."

A Department of Health spokesperson said: "NICE does not recommend the use of lumacaftor and ivacaftor (Orkambi) for treating cystic fibrosis and has published guidance to this effect (TA398).

"In August 2016, the department endorsed TA398 as applicable here, therefore Orkambi is not offered as a routinely commissioned treatment for cystic fibrosis."

A government spokesman said: "Cystic fibrosis is a life-limiting condition and it is absolutely right that patients should have access to cost-effective, innovative medicines and technologies on the NHS. However, Orkambi has not been approved by the healthcare regulatory bodies in England, Scotland or Northern Ireland."

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