The National Institute for Health and Care Excellence approves the use of gene-editing treatment for blood disorders including sickle cell disease.
The National Institute for Health and Care Excellence (NICE) has approved a gene-editing treatment for use with NHS patients over the age of 12 with a severe form of sickle cell disease.
The gene therapy, known as exa-cel or Casgevy (exagamglogene autotemcel) is based on CRISPR gene editing. It was developed through a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics.
“The treatment is an example of true medical innovation and will provide patients with no other options a potential cure for the painful, debilitating symptoms of their diseases. It also offers promising research avenues for other genetic diseases,” said Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust. The Trust led clinical trials as part of an international collaboration between academic researchers, industry partners and patients.
Both sickle cell disease and beta thalassaemia are genetic conditions caused by errors in the genes for haemoglobin, which are essential for carrying oxygen to all organs and tissues of the body. Sickle cell disease can cause severe pain, organ damage and shortened life span due to misshapen or “sickled” blood cells. Around 15,000 people in the UK have sickle cell disease and it is particularly common in people with African or Caribbean family backgrounds.
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.
To date, the only long-term cure option for the two blood conditions is a bone marrow transplant but optimal outcomes are only achieved from a closely matched donor. Transplants also carry a risk of rejection and complications and are only available to a small fraction of people living with the conditions.
The treatment will be offered at specialist NHS centres in London, Manchester and Birmingham, and will be available for eligible patients over the age of 12 who experience recurrent sickle cell crises and would be suitable for a stem cell transplant but where a donor is not available.
It is estimated that around 50 such patients will receive the treatment each year. A similar number of children and adults with sickle cell disease receive a stem cell transplant on the NHS each year.
