A revolutionary therapy for sickle cell disease will soon be offered to NHS patients, in a breakthrough that Prof Owolabi, of NHS England, called a “monumental step forward”.
Starting January 31, the one-time treatment known as exagamglogene autotemcel (‘exa-cel’) will be offered to around 50 patients every year, for which the results could be life-changing.
Sickle cell disease is an inherited blood disorder that results in an abnormality in the haemoglobin found in red blood cells. It can result in life-threatening cases and cause recurring intense pain, when blood vessels become blocked by misshapen red cells. About 15,000 UK residents suffer from SCD, with 250 new cases each year.
Exa-cel utilises Nobel Prize-winning CRISPR technology to edit a gene within the patient’s stem cells so that the body produces functioning haemoglobin. Blood stem cells are removed from a patient’s body and edited in a laboratory using CRISPR. The treated cells are then returned to the patient through an infusion.
The total price of the treatment for all will come to £1.65 million. However, NHS England has negotiated a deal for a reduced price for taxpayers, allowing the treatment to be offered through the Innovative Medicines Fund, a funding program for new medicines.
NHS chief executive, Amanda Pritchard, said: “It is just the latest in a series of revolutionary gene therapies NHS England has secured for patients, and we are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.”
“Sickle cells” by Dr Graham Beards is licensed under CC BY-SA 3.0.