How this revolutionary drug is helping mum Sammie see milestones she thought she might miss
PUBLISHED: 07:30 03 July 2020
A Suffolk mum who was one of the first people in the UK to receive a game-changing drug to treat her cystic fibrosis says she is delighted it is now to become available on the NHS in England.
Sammie Read, of Mendlesham, said the decision this week to fund Kaftrio would transform the lives of thousands of people with the condition.
Cystic fibrosis (CF) is a genetic mutation which causes a build-up of thick sticky mucus in the lungs, digestive system and other organs. People with the condition seldom live beyond middle age.
But following the deal struck by NHS England with the drug’s manufacturer Vertex Pharmaceutical, Kaftrio will become widely available later this year.
Sammie, aged 40, was one of the first UK users in a trial earlier this year. She said: “I was given the drug on compassionate grounds and it transformed my life.
“So I’m absolutely delighted that so many people with CF are going to be able to live a natural life span rather than with the uncertainty they had before.”
Kaftrio is a combination of three drugs - ivacaftor, tezacaftor and elexacaftor - and, while it is not expected to help all CF sufferers, it will help up to 90% of them.
It is available to those over the age of 12 and with either two copies of the F508del gene mutation, or one copy of F508del and one of a ‘minimal function mutation’.
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Sammie has the F508del mutation and another mutation so rare that there is only one other known sufferer in the world.
She was put on a trial while being treated at the Royal Papworth Hospital in Cambridge for five weeks at the beginning of the year suffering from a partially collapsed lung.
Within days her lung function began to recover, improving by 10% after just two weeks.
The successful drug trial has meant Sammie will enjoy major milestones in her life such as her son Bradley, now 19, buying and moving into his first home in Stowmarket.
She and her husband are hoping to be able to visit Bradley for a housewarming once the risk from Covid-19 reduces as they have been shielding since March.
Over the years Sammie has fundraised for the Cystic Fibrosis Trust and at her 40th birthday ball last year she raised approximately £20,000.
She has also started a YouTube channel to document her life during lockdown, and has shared updates on how the drug trial is improving her health.
David Ramsden, chief executive of the CF Trust, said: “It is fantastic news that a deal has been done between NHS England and Vertex Pharmaceuticals and Kaftrio will now be available to thousands of people across England in the coming weeks. This will truly save lives.
“This is a great day, but we know there is more to do and we will not stop until everyone with CF across the UK has access to life saving drugs.”
For more details go to the Trust website.
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