Thousands of people on the NHS are set to benefit from a ‘groundbreaking’ sickle cell gene therapy.
The National Institute for Health and Care Excellence has approved a one-off gene therapy known as exagamglogene autotemcel (exa-cel) to stop painful and unpredictable sickle cell disease symptoms in older children and adults.
NHS chief executive, Amanda Pritchard, called this a ‘leap in the right direction’.
‘Before the availability of this new one-time treatment, the disease had required lifelong treatment regimens, greatly impacting on quality of life, with people suffering from chronic pain, fear of sickle cell crises, hospitalisation, and shortened life expectancy, and many patients describe it as ‘looming over their daily lives.
‘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.’
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About 15,000 people in England live with the condition, which mainly affects people of Black African and Black Caribbean backgrounds. In sickle cell disease, a gene mutation causes red blood cells to become irreversibly sickle shaped, rather than flexible smooth discs. This can lead to haemolytic anaemia, a blood condition that occurs when red blood cells are destroyed faster than they are replaced. Over a long period, the disorder can cause severe organ damage and intense pain if damaged red blood cells block vessels and restrict oxygen supply, which can also lead to strokes and heart failure.
Exa-cel, also known as Cagevy and manufactured by Vertex, uses CRISPR technology to edit a person’s gene in their bone marrow stem cells. The treated stem cells are then transfused back into the patient’s body, enabling the production of functioning haemoglobin. In clinical trials, all patients who received exa-cel avoided hospitalisation for a year following treatment and almost 98% avoided hospitalisation around 3.5 years later.
The gene therapy will be available in specialist centres in London, Manchester and Birmingham to people aged 12 and over who get recurrent sickle cell crises and who cannot find a donor for a stem cell transplant. While the list price of the treatment is £1.65 million, NHS England has struck a deal to access the treatment at a reduced price for taxpayers. It is set to be offered through the Innovative Medicines Fund that fast-tracks funding for new medicines.
John James OBE, chief executive officer of the Sickle Cell Society, welcomed the ‘groundbreaking’ gene therapy treatment.
‘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. We remain dedicated to our commitment to advocating for improved treatment options and better outcomes for all those affected by this condition. For now, we take a moment to celebrate this historic milestone, a turning point in the fight against sickle cell that promises to change lives for the better.’