‘Ground-breaking’ gene treatment for sickle cell disease to be rolled out on NHS
A GROUNDBREAKING genetic treatment with the potential to cure sickle cell disease will be rolled out on the NHS.
The one-off treatment costs more than £1million per patient but may essentially wipe out the illness in 97 per cent of people.
Sickle cell disease affects about 15,000 Brits, most commonly black people, and 50 severe cases per year are expected to be eligible.
It is a genetic condition that causes blood clots as it makes red blood cells form incorrectly.
The treatment uses cutting-edge Crispr gene editing to remove blood from the patient, correct faulty DNA and then pump it back into the body.
This then triggers the production of healthy red blood cells and prevents symptoms of the disease.
‘Hope of a cure’
It costs £1.6million per patient on the open market but the NHS will get a hefty discount.
NHS chief executive Amanda Pritchard said: “This is a leap in the right direction and offers hope of a cure for people facing a severe form of the disease.”
John James, chief of the Sickle Cell Society, said: “We are absolutely thrilled and the significance of this milestone for the sickle cell community cannot be understated.
“Today’s result will give hope to many and is a turning point in the fight against sickle cell that promises to change lives for the better.”
Clinical trials have found the treatment can stop sickle cell crises, the most serious symptoms where blood vessels become painfully blocked.
There were almost 14,000 hospital admissions for such symptoms in 2024 – almost half of all the 32,000 admissions for sickle cell disease.
Patients could also be protected from infections and other effects of the disease such as high blood pressure and kidney issues.
It will be offered by specialist clinics in London, Manchester and Birmingham to patients over the age of 12 who have severe enough illness to qualify for a stem cell transplant but who do not have a donor.
WHAT IS SICKLE CELL DISEASE?
SICKLE cell disease is a genetic condition that prevents red blood cells from developing properly.
The cells can become abnormally shaped and die quicker than healthy blood cells, raising the risk of clots or blood vessel blockages.
Most people live normal lives with the condition but it can trigger flare-ups called sickle cell crises, when symptoms become painful and may lead to complications.
Signs include:
- Dizziness
- Pain
- Tiredness and weakness
- Headaches
- Shortness of breath
Patients with sickle cell disorders are also at higher risk of infections, anaemia (low blood iron), gallstones, stroke, high blood pressure and kidney problems.
There is no definitive cure for sickle cell disease and many patients require treatment throughout their lives.
An estimated 15,000 people in England have it and it is more common in black people.