Hope For English Thalassaemia Patients

Thalassaemia is an inherited condition. It causes the body to produce too little haemaglobin. In the UK, it mainly affects people from a Mediterranean, Asian or Middle-Eastern background. Folk with severe transfusion-dependent beta thalassaemia, suffer painful side-effects and rarely live past the age of 50. They now, however, can be given treatment with Casgevy. Casgevy is a one-off gene therapy. It edits the patient's bone marrow stem cells so they produce functional haemaglobin (https://www.theguardian.com/society/article/2024/aug/08/nhs-to-offer-life-changing-gene-therapy-for-blood-disorder-thalassaemia).International clinical trials found that more than 90% of beta thalassaemia patients getting Casgvy treatment, needed no blood transfusion for at least a year. The National Institute for Health and Care Excellence (NICE) does a cost-benefit analysis of all new drugs and medical treatments. It has approved the NHS offering Casgevy to about 450 patients. Treatment is normally very expensive but NICE have agreed a special deal with Casgevy's manufacturers. It's hoped that this gene-editing treatment will greatly reduce the need for blood transfusions and allow these thalassaemia patients to live longer.