Baby Walter Olney and his family add their voices to plea for specialist medicines to be free on 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 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 around 1,000 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 as he grows up.
The medication which has been shown to help those with the disease, which is not only debilitating but also life-shortening, is considered too 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 newborns.
“I was contacted by the nurse. She said could she come over and see me. She also wanted me to get Charlie from work. I knew that cystic fibrosis was the most likely thing for Walter.
“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, 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 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.”
In June 2016 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.
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.