A groundbreaking £1.65m gene therapy treatment has been approved for use on the NHS in England, offering a potential cure for people living with severe sickle cell disease.
The treatment, known as exagamglogene autotemcel (exa-cel), is a one-time gene therapy that edits the faulty gene in a patient’s own stem cells. This innovative approach has shown significant promise in clinical trials, with 96.6% of participants experiencing a “functional cure.”
The NHS estimates that approximately 50 patients per year will be eligible for this treatment, specifically those with severe sickle cell disease who are suitable for a stem cell transplant but lack a matched donor. This treatment has the potential to transform the lives of these individuals, who often experience debilitating and painful sickle cell crises.
Sickle cell disease is a genetic disorder that affects the shape of red blood cells, causing them to become crescent-shaped and hindering blood flow. This can lead to severe pain, organ damage, and even premature death. In England, approximately 17,000 people are living with sickle cell disease, with around 4,000 estimated to be eligible for this new treatment.
The approval of exa-cel is a significant milestone in the fight against sickle cell disease. Campaigners and experts have welcomed this decision, hailing it as a “monumental step forward” and “groundbreaking.”
Prof Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England, emphasized the significance of this treatment, stating that it represents a “very real prospect of a cure” for severe sickle cell disorder.
John James OBE, Chief Executive of the Sickle Cell Society, expressed his thrill at the approval, highlighting the importance of this milestone for the sickle cell community.
Yasmin Sheikh, Head of Policy and Public Affairs at Anthony Nolan, also welcomed the decision, noting that it represents a “leap forward” in the treatment of this debilitating and life-threatening condition.
The approval of exa-cel is a testament to the power of innovative research and collaboration. As this treatment becomes available on the NHS, it is expected to bring new hope to those living with severe sickle cell disease, offering a potential cure and improved quality of life.
