Casgevy, developed by Vertex, has been approved by the National Institute for Health and Care Excellence (NICE) for use on the NHS in England. This therapy has been recommended for patients over 12 years of age with beta thalassaemia who meet specific criteria.
People with beta thalassaemia struggle to produce enough haemoglobin, which is the protein in red blood cells that carries oxygen around the body. Casgevy involves gene-editing, using Nobel Prize winning Crispr-technology, in which stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient’s body. As part of the process a course of chemotherapy is needed in order to kill the old stem cells that were producing broken haemoglobin. Fortunately, gene-editing is only intended to be done once so any unpleasant aspects shouldn’t need to be repeated.
NICE’s committee determined that Casgevy “could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions,” NICE’s director of medicines evaluation, Helen Knight, explained in a press release.
Reactions about this new treatment have been very positive. Amanda Pritchard, the NHS chief executive, said: “This is a historic moment for people living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS”. Whilst, Romaine Maharaj, executive director of the UK Thalassaemia Society, said “we stand on the brink of a revolutionary breakthrough” and “it is a beacon of hope”.
For more info, please click this NHS link: bit.ly/4cdpKkJ
