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The NHS in England has approved a gene-editing therapy worth £1.65m for those suffering from sickle cell disease. Experts predict around 50 people a year with the blood disorder will receive the one-off treatment, which is known as Casgevy or Exa-cel. While campaigners and experts describe the therapy as groundbreaking, it is worth noting that it will only be given to those with recurrent sickle cell crises aged 12 or over who cannot access a donor for a stem cell transplant. There are approximately 15,000 people in England with the condition, which is often present in people with Black Caribbean and Black African heritage.
The gene therapy works by editing a specific gene and allowing the body to produce more healthy red blood cells. In trials, all patients who received the therapy avoided stays in the hospital for a year after treatment, with most doing so for three-and-a-half years. Its availability on the NHS has been described as a milestone and has given hope to people with sickle cell disorder. Some have called it a life-changing moment for the community.
Patients will require conditioning chemotherapy to ensure the body can receive edited stem cells. While the full treatment can have side effects, including headaches and bleeding problems, its benefits provide a very real prospect of a cure. It is to be performed at specialist centres in London, Birmingham, and Manchester.
The only other treatment option for a cure is a stem cell transplant. This is only possible if there is a closely matched donor available and has a risk of rejection. The gene therapy has been approved for another inherited blood disorder, beta thalassemia, and is available in countries such as Germany, France, and Italy. The NHS reached a confidential agreement with manufacturer Vertex over payment
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