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, from today (August 8), 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.
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.”
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 per cent 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.
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.
Beta thalassaemia is an inherited blood disorder that affects the red blood cells, which carry oxygen around the body.
It mainly affects people of Mediterranean, south Asian, southeast Asian and Middle Eastern background.
There are around 2,300 people with thalassaemia in the UK, with an estimated 800 people with the severe form of the condition that rely on regular blood transfusions. In the UK, the largest groups affected are of Pakistani, Indian and Bangladeshi ethnicity.
The disorder requires lifelong treatment and can greatly impact on quality of life, with people suffering from anaemia, chronic pain and many also reporting psychological impacts, such as anxiety and depression.
Beta thalassemia is a life-limiting condition, with many patients not expected to live beyond their 50s.
Previously, the only curative treatment for patients has been a stem cell transplant, but this is only available to a small number of people – it can be difficult to find matched donors and people’s bodies can reject transplants.
Romaine Maharaj, Executive Director, UK Thalassaemia Society, said: “With NICE’s approval of gene therapy for transfusion dependent thalassaemia under the NHS managed access scheme, we stand on the brink of a revolutionary breakthrough.
“This transformative treatment offers patients a life-changing opportunity, enabling them to repair their own cells and embrace a future free from the challenges of their condition.
“It is a beacon of hope that underscores the power of innovation in medicine, paving the way for curative options that can truly enhance the quality of life for everyone affected.”
