Sickle cell patients offered gene-editing therapy
The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’), has been approved for use on the NHS in England by the National Institute for Health and Care Excellence NICE for older children and adults with a severe form of sickle cell disease. Private GP in London Dr Martin Harris is interested for his patients to employ this revolutionary therapy.
In England, there are around 15,000 people living with sickle cell disease – an inherited blood disorder, with 250 new cases a year. It mainly affects people from Black African and Black Caribbean backgrounds. 32,000 hospital admissions in England in 2023-24 for sickle cell disorders. Almost 14,000 of these admissions were admissions for sickle cell anaemia crises.
In sickle cell disease, a gene mutation causes red blood cells to become irreversibly sickle shaped, which can lead to haemolytic anaemia – a blood condition that occurs when your 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.
Clinical trials suggest exa-cel can stop painful and unpredictable sickle cell crises – the most common symptom of sickle cell disease – where blood vessels become blocked causing severe pain, with experts saying the therapy offers patients a chance of disease-free life. Researchers concluded there was a ‘functional cure’ in 96.6% of exa-cel trial participants that received it.