Sickle cell disease can cause painful and unpredictable episodes (Image: Getty)
A revolutionary gene therapy treatment costing a staggering £1.65 million has been approved for use, offering patients with an inherited blood disorder hope of a cure.
Sufferers of sickle cell disease experience agonising and unpredictable sickle cell crises, where blood vessels become blocked causing severe pain.
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.
Blood stem cells are removed from the patient’s body and edited in a laboratory using Crispr. They are then infused back into the patient, prompting the body to begin producing functioning insulin.
The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell .
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Around 50 people a year are expected to benefit from the expensive one-off treatment.
It has a list price of £1.65 million but the NHS have negotiated a confidential pricing arrangement with pharma company Vertex.
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.”
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.
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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 National Institute for Health and Care Excellence said the treatment could 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.
It will be offered at specialist NHS centres in , Manchester and Birmingham.
NICE chief executive Dr Samantha Roberts added: “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.”
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.