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New drug is set to give Katie fresh lease of life

By Exeter Express and Echo  |  Posted: December 27, 2012

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A TEENAGER from Exeter has been given the best Christmas present – a new drug that could save her life.

Katie Murphy, 14, who suffers from cystic fibrosis, is currently unable to live a normal life due to the amount of medication she needs to take every day.

Previously her only option for a better life had been a lung transplant. But last week the Government announced it was to fund a new drug Kalydeco that Katie and her family have been campaigning for over many years.

Her mother Denise, who lives in St Leonard's, said: "This is an absolutely fantastic day and we are all so excited. For Katie it means she can go on sleep-overs without dragging loads of equipment. She will also be able to go to school every day and not get behind. From January she will only have to take two tablets of Kalydeco every day, which will change her life."

Katie took part in a medical trial of the drug last year but despite the positive results that saw her lung function dramatically improve, the drug was not made available on the NHS because of licensing issues and its price tag, which was put at £182,000 per patient per year.

The new drug is the first available that targets the underlying cause of cystic fibrosis — a faulty gene and its protein product, CFTR.

Mrs Murphy added: "Previously the only hope for people with Katie's condition was to have a lung transplant. But this state-of-the-art drug could save her life and others like her. It is not yet licensed for children under six but it needs to be given out as young as possible to prevent lung damage."

During the one-month trial Katie had to take a tablet twice a day. Her weight went up by 4kg and lung function increased by at least 10 per cent.

"Her quality of life at the moment is very challenging but this drug will improve her life immeasurably," Mrs Murphy added. "It is like a holy grail for cystic fibrosis sufferers.

"If I look at it dispassionately the cost of Katie's care on the NHS over however many years she is going to live, is equal or more to giving her the drug now.

"But looking at it as a parent I just want my daughter to have the best chance in life possible."

Katie, a pupil at St Peter's School, has a form of cystic fibrosis called a gating mutation, in which the defective protein moves to its proper place at the surface of the cell but does not function correctly.

The CFTR protein instead acts like a locked gate, impeding the proper flow of salt and fluid in and out of the cell. The aim of Kalydeco is to unlock that gate and help restore the function of the defective protein.

Katie is currently on around 60 drugs and at least four nebulisers to manage her condition. On the Government's announcement, she said: "This is brilliant. I will be able to do so much more and be a lot freer – instead of carrying tons of medicine and treatments around."

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