A groundbreaking gene-editing therapy for sickle cell disease, Casgevy (Exa-cel), will be offered on the NHS in England at a negotiated price below its £1.65m market cost. Around 50 patients per year are expected to receive it.
This one-time treatment uses Crispr gene-editing to modify blood stem cells, allowing the body to produce healthy fetal haemoglobin, which prevents red blood cells from becoming sickle-shaped. Trials have shown significant success, with patients avoiding hospital stays for at least a year after treatment.
The process involves:
- Extracting blood stem cells from the patient.
- Editing a gene to restore fetal haemoglobin production.
- Conditioning chemotherapy to prepare the body.
- Reintroducing modified stem cells, which multiply and create stable red blood cells.
Sickle cell disease, which mainly affects Black African and Caribbean individuals, causes severe pain and life-threatening complications. NHS leaders and campaigners hail this as a monumental step forward, offering hope for a potential cure despite the intensive treatment process and possible side effects.