£1.65m gene-editing therapy offers hope of cure for some blood disorder patients

A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder hope of a cure.
Campaigners reacted with joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder.
It is thought that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition.
NHS officials estimate that around 50 people a year will receive treatment.
Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020.
Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications
Dr Samantha Roberts, Nice
It works by editing the faulty gene in a patient's own stem cells.
Cells are taken from a patient to a laboratory where the Crispr technology is used. The edited cells are then infused back into the patient, which prompts the body to produce healthy red blood cells.
The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant.
But it is not given to many patients due to the risks involved.
Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found.
In March last year, Nice rejected Casgevy for people with severe sickle cell disease.
At the time Nice said that it needed further detail about the effectiveness of the treatment, also known as exagamglogene autotemcel (exa-cel) and made by Vertex.
It also needed a commercial agreement for the drug, which has a list price of £1.65 million.
Now health officials have reached a confidential agreement with Vertex on how much the NHS will pay for the treatment.
Nice said that the treatment can be offered under a 'managed access scheme' for treating severe sickle cell disease in some people 12 years and over with 'recurrent vaso-occlusive crises' who have a certain genotype.
'Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing Nice's aim of reducing health inequalities associated with the condition and getting the best care to patients fast,' said Nice chief executive Dr Samantha Roberts.
Ludovic Fenaux, senior vice president at Vertex International, added: 'Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease.'
There are around 17,500 people with sickle cell disease in the UK.
It is particularly common in people with an African or Caribbean family background.
Symptoms can include very severe pain, serious and life-threatening infections and anaemia.
Mehmet Tunc Onur Sanli, 42, from London, who was diagnosed with sickle cell disease aged 11, said: 'Because of my illness, I often experience pain in my chest, bones, and muscles.
'I had surgery on my spleen when I was six and a hip replacement at 22 – I will probably need another hip replacement in the next few months or years.
'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.
'The pain is the worst I have ever felt in my life – it's hard to put into words.
'Not having to go to hospital for regular transfusions or taking medicine anymore would be a dream to me – gene therapy could offer that – but there's still a lot to consider in terms of the side effects that could come with this treatment and whether it would be the right choice for me.'
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.
NHS England chief executive Amanda Pritchard said: 'This is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition.
'This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them.
'We are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.'
John James, chief executive of the Sickle Cell Society, said: 'We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today.
'The significance of this milestone for the sickle cell community cannot be understated – today's result will give hope to many and is the result of determined campaigning.'
The Medicines and Healthcare products Regulatory Agency (MHRA) approved the treatment in November 2023 for patients aged 12 and over after a 'rigorous assessment of its safety, quality and effectiveness'.
It was the first regulator in the world to approve the treatment.
Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: 'This groundbreaking decision to fund the UK's first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition.
'Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure.
'With Casgevy, we now have a treatment that offers hope to more patients, the majority of whom are from African and African-Caribbean backgrounds and have experienced years of feeling ignored.
'This treatment has the potential to transform lives and offers a glimpse into the exciting possibilities of gene therapy.'
Nice approved the treatment for certain patients with a severe form of the blood disorder thalassemia in August last year.
The NHS spending watchdog said that it will collect more data while patients receive the treatment on the NHS before it evaluates the medicine again.
It said that collecting more data through a managed access agreement may resolve some uncertainty in the evidence, particularly about how long the benefits of treatment with exa-cel last.
Health minister Andrew Gwynne said: 'By offering a treatment that could allow patients to live a life free of debilitating illness, we will give people with conditions like sickle cell disease more freedom and independence, all whilst protecting vital NHS emergency services.'
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 ground-breaking 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.'

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