A “groundbreaking” £1.65 million treatment has been approved for use on the NHS, offering patients with severe sickle cell disease some hope of a cure.
It is believed around 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for some patients with the genetic condition.
Around 50 people a year will receive the treatment, NHS officials estimate.
Professor Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust – which led the UK arm of the clinical trials for exa-cel, said: “Together with patients and industry partners, we are proud to be part of the groundbreaking research and international academic collaboration that has made this treatment possible.
“The treatment is an example of true medical innovation and will provide patients with no other options a potential cure for the painful, debilitating symptoms of their diseases.
“It also offers promising research avenues for other genetic diseases.”
For sickle cell patients like Lanre Ogundimu, the drug could be a game-changer. In 2018, she suffered a stroke, a pulmonary embolism and a blood transfusion reaction as a result of the disorder.
She spent 30 days in hospital, six months in physiotherapy, more than 12 months in therapy and nine months not working.
“I felt weak all the time,” she said. “This was the lowest point in my life. It impacted my freedom and independence, my income, my career trajectory, and my ability to contribute towards society.”
Sickle cell disease is the name for a group of inherited conditions that affect the red blood cells – the most serious type is called sickle cell anaemia.
The disorder causes red blood cells to take on a “sickle” shape, break and die early, which vastly reduces the amount of oxygen they can carry around the body.
It is particularly common in people with an African or Caribbean family background.
It is caused by a gene that affects how red blood cells develop – if both parents have the gene, there is a one in four chance of each child being born with the disease. Having the gene does not necessarily mean parents have the disease but are carriers of the sickle cell trait.
The main symptoms are painful episodes (called sickle cell crises) which can last for days or weeks, an increased risk of serious infections, and anaemia, which can cause tiredness and shortness of breath.
Currently, the only cure for sickle cell disease is a stem or bone marrow transplant – these are not done very often because of the risks involved.
Mehmet Tunc Onur Sanli, 42, from London, was diagnosed with the disorder aged 11. He had surgery on his spleen when he was six and a hip replacement at 22.
“I also suffer from regular sickle cell crises – last year, I had to go to the hospital at midnight after waking up in severe pain, and overall, I had to visit the hospital five or six times due to crises,” he said.
“The pain is the worst I have ever felt in my life – it’s hard to put into words.”
While he says there is still “a lot to consider” with the new treatment, not having to go to hospital regularly “would be a dream for me”.
How does the treatment work?
Casgevy – also known as exa-cel – earned its inventors the Nobel Prize for chemistry in 2020.
It works by editing the faulty gene in a patient’s own stem cells. It is an option for patients when a stem cell transplant is suitable but no donor can be found.
The treatment was first rejected by Nice in March last year – it said it needed further detail about its effectiveness. It also needed a commercial agreement for the drug, which has a list price of £1.65m.
It is not known how much the NHS will pay Vertex for the treatment, as health officials reached a confidential agreement with the drug company.
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In clinical trials, all patients who received exa-cel also avoided hospital admission for a year following treatment – and almost 98% had still avoided being admitted to hospital around 3.5 years later.
The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham.
‘Absolutely transformative’
NHS England chief executive Amanda Pritchard said the NHS would be funding the new treatment “straight away”.
“This is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition,” she said, adding that it offered a hope of a cure for patients facing a severe form of the disease.
“[It] could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them.”
University student Funmi Dasaolu has experienced chronic fatigue and pain her entire life.
The 31-year-old, from Oxfordshire, has been admitted to hospital many times due to the condition – in 2022 she was admitted to hospital seven times, and for the last five years has been receiving regular blood transfusions.
“Today is a momentous day for those living with or affected by sickle cell disorder,” she said.
“After months of campaigning, I’m overjoyed and so very grateful exa-cel has been finally approved.
“It will be truly transformative for patients and offers us the chance of a life without this terrible condition. A chance to grow old, to fulfil our dreams and to live a pain-free life.”
