Baby Walter Olney from Manea and his family write to Prime Minister Theresa May to call for precision medicines to be available on the NHS
A four-month-old baby and his family are among those petitioning the Government to make new precision medicines available on the NHS.
Walter Olney, from Manea, was diagnosed with Cystic Fibrosis at just 13 days old and his friends and family have written emotional and heartfelt letters to Prime Minister Theresa May asking for new medicines that could prolong and enhance his life to be made available on the NHS.
Their letters were among approximately 1,000 letters were delivered by Gemma Weir, who has helped spearhead the campaign, and her daughter Ivy to 10 Downing Street on Wednesday.
Walter's mum Leanne explained Walter, who has the most common mutation of the genetic disease, is too young to benefit from specialist medicine right now but they could help him as he grows up.
However, the medication which has been shown to help those with the disease, which is not only debilitating but also life-shortening, is considered to expensive to be freely available on the NHS and is currently only prescribed on compassionate grounds.
"I take that to mean that it is when someone is coming to the end of their life," explained Leanne, who with husband Charlie was given the devastating news about Walter after he had the routine heel prick test given to all new borns.
"I was contacted by the nurse who said something had come up in Walter's heel prick test and could she come over and see me right now, she also wanted me to get Charlie home from work. I knew anything that shows up in the test is bad and I also knew that cystic fibrosis was the most likely thing for Walter.
"It has been a real learning curve. Walter has to take medication multiple times a day including a special enzyme before every feed and he also has to have daily sessions of physiotherapy including PEP (positive expiratory pressure therapy) which helps clear his lungs and also other exercises to help strengthen his lungs," said Leanne.
Cystic fibrosis or CF as it is known is an inherited disease caused by a faulty gene. This gene controls the movement of salt and water in and out of your cells, so the lungs and digestive system become clogged with mucus, making it hard to breathe and digest food.
There are around 10,400 people with the disease in the UK and while there are conventional treatments that target the symptoms precision medicines tackle the underlying genetic mutations which cause the condition.
One of these is Orkambi, a precision medicine that nearly half of people with cystic fibrosis, including children aged six and over, could benefit from in the UK.
Leanne said: "Orkambi is not a cure, but it has been found to slow decline in lung function – the most common cause of death for people with cystic fibrosis. It has also been shown to reduce chest infections requiring hospital treatment by up to 61 per cent. People say it is expensive it costs around £104,000 a year to treat one person, but it cuts the need for other medication and helps prevent people from becoming sick and needing to go into hospital, so it could save money in the long run."
Orkambi received its European license in November 2015 and in June the following year the UK Government agency the National Institute of Health and Care Excellence (NICE) recognised Orkambi as an important treatment.
They did not recommend the drug, though, on grounds of cost-effectiveness. This means it is unavailable to most people with cystic fibrosis across the UK.
Two million people in the UK are carrying the faulty gene without realising it. Both Leanne and Charlie carry the same faulty gene but only discovered this after Walter was diagnosed they have been told they have a one in four chance of any other children having the disease.
Half of people with cystic fibrosis alive today are expected to live into their forties, thanks to earlier diagnosis and ongoing developments in care and treatments
For confidential advice, support and information on any aspect of cystic fibrosis, including help with financial support contact the Cystic Fibrosis Trust helpline on 0300 373 1000 or 020 3795 2184.