NHS patients living with a genetic blood disorder are set to be among the first in the world to benefit from a “life-changing” gene-editing treatment which offers hope of a cure.
The one-off gene therapy, Casgevy, has been approved by for use on the NHS in England by the National Institute for Health and Care Excellence (NICE) for older children and adults with a severe form of thalassaemia.
The NHS in England is among the first healthcare systems in the world to offer the treatment, with an estimated 460 patients in England currently living with transfusion-dependent beta thalassaemia, aged 12 and older, potentially eligible for the therapy which uses gene-editing CRISPR technology.
It will be offered at seven highly specialist NHS centres across the country within weeks, with the therapy being manufactured in the UK.
Thalassaemia is the name for a group of inherited conditions that affect a substance in the blood called haemoglobin. Most patients who could be eligible for this treatment currently need transfusions every 3-5 weeks to survive, which have a major impact on their quality of life.
In international clinical trials, 93% of patients with beta thalassaemia did not need a blood transfusion for at least a year after having the treatment, and it is hoped that the therapy could be a lifetime cure.
The therapy, Casgevy (exagamglogene autotemcel), works by editing a gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin – it is the first approved therapy to use the Nobel Prize-winning CRISPR technology.
Amanda Pritchard, 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.
“Ordinarily, patients experience painful side effects and undergo regular transfusions which severely impact their quality of life, but this therapy offers people a life free from that as well as the hope of living longer, which is truly amazing news.
“This is the latest in a series of revolutionary gene therapies to be secured by NHS England over the past five years, bringing significant benefit for patients – and thanks to funding through our Innovative Medicines Fund, this one-off therapy will be fast-tracked to patients who could benefit from the new lease of life it promises.”
Blood stem cells are removed from a patient’s body, they are edited in a laboratory using CRISPR technology, and the treated cells are then returned to the patient via an infusion.
Patients also need to receive chemotherapy before treatment with Casgevy. This is followed by a 4-6 week stay in hospital to allow the treated stem cells to embed themselves in the bone marrow and begin producing healthy red blood cells.
