Stem cell charity Anthony Nolan welcomes the decision from NICE (the National Institute for Health and Care Excellence) to recommend the CRISPR gene therapy, exa-cel (exagamglogene autotemcel), as a treatment for sickle cell disorder.
Exa-cel, also known by the brand name Casgevy, involves modifying a patient’s own stem cells to prompt the production of healthy red blood cells, addressing the symptoms of sickle cell.
The treatment was initially rejected by NICE in a draft decision in March 2024, but following extensive consultation with charities, patient groups and NHS England, this has been overturned.
Sickle cell can be a debilitating condition as patients often suffer from acute pain episodes known as ‘crises’. Crises are a physical, emotional, and mental burden to patients. Sickle cell also has a chronic effect on almost every major organ system, impacting life expectancy and day-to-day quality of life.
Historically the only curative treatment available to people with sickle cell in the UK was a donor stem cell transplant – however not all patients have a matching donor available. Exa-cel could provide a ‘functional cure’ for anyone unable to have a stem cell transplant.
Unlike a stem cell transplant, which uses cells from a healthy donor, exa-cel involves genetically editing the patient’s own cells which are re-infused into them.
Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said: "This groundbreaking decision to fund the UK's first ever CRISPR-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition.
“Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure.
“With Casgevy, we now have a treatment that offers hope to more patients, the majority of whom are from African and African-Caribbean backgrounds and have experienced years of feeling ignored.
“This treatment has the potential to transform lives and offers a glimpse into the exciting possibilities of gene therapy."
People who live with sickle cell have expressed their delight following the news.
Funmi Dasaolu, 31 from Oxfordshire, has experienced chronic fatigue and pain her entire life, which has severely impacted her education and social life. She has been hospitalised many times – in 2022 she had seven hospital stays in just one year. For the last five years she has been receiving regular blood transfusions to help with her symptoms. Funmi said:
“Today is a momentous day for those living with or affected by sickle cell disorder. After months of campaigning, I’m overjoyed and so very grateful exa-cel has been finally approved. It will be truly transformative for patients and offers us the chance of a life without this terrible condition. A chance to grow old, to fulfil our dreams and to live a pain free life.”
As a child Toby Bakare, 35 from South London, frequently missed school due to painful sickle cell crises and was hospitalised four to six times a year. Throughout adulthood crises were less frequent for Toby but more painful and difficult to manage, impacting all areas of his life, including his career and relationships. Because of this, Toby made the decision to have a stem cell transplant and was fortunate to have a sibling who could be his donor. Toby said:
“This decision is a game changer for the thousands of people like me living with this disorder. They now have a chance to live without pain, fatigue and all the other symptoms of sickle cell which can make quality of life so poor.”
Exa-cel will be rolled out by pharmaceutical company Vertex and will be available through the NHS to patients with sickle cell disorder in England and Wales. Its effectiveness, safety and usage will be continually monitored to ensure it meets expected standards and provides value for the NHS.