UK Health System to Offer Cutting-Edge Gene Therapy for Sickle Cell Disease
NHS to Provide Groundbreaking Treatment
The UK’s national health service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care Excellence (NICE) announced on Friday.
Cost and Availability
The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the state-funded healthcare system around £1.65 million (US$2.1 million) per course. The treatment is expected to be suitable for around 50 patients per year.
Regulatory Approval
The UK’s MHRA medical regulator in 2023 became the first in the world to approve the therapy, which uses the gene-editing tool CRISPR.
What is Sickle Cell Disease?
Sickle cell disease is a serious and lifelong condition caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.
How Does the Therapy Work?
The therapy involves taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory and then infusing them back into the patient.
Similar Treatment for Beta Thalassemia
The same technology was approved for use in the NHS in August to treat beta thalassemia, another rare blood disorder.
Conclusion
The UK’s decision to provide this cutting-edge gene therapy marks a significant milestone in the treatment of sickle cell disease. The therapy offers new hope for patients and their families, and the NHS’s commitment to providing this treatment is a testament to its dedication to delivering the best possible care.
FAQs
Q: What is the cost of the therapy?
A: The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the NHS around £1.65 million (US$2.1 million) per course.
Q: How many patients will be treated with this therapy?
A: The treatment is expected to be suitable for around 50 patients per year.
Q: What is the gene-editing tool used in this therapy?
A: The therapy uses the gene-editing tool CRISPR.
Q: What is sickle cell disease?
A: Sickle cell disease is a serious and lifelong condition caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.