Groundbreaking Gene Therapy for Sickle Cell Approved by NHS
A revolutionary gene therapy for sickle cell disease has been approved for use by the NHS in England, marking a major breakthrough in treatment for the debilitating condition. The one-time treatment, known as exagamglogene autotemcel (exa-cel), is designed to edit faulty genes in a patient’s stem cells, offering a potential cure.The National Institute for Health and Care Excellence (NICE) initially withheld approval last year, citing concerns over its long-term effectiveness. However, following extensive clinical trials, which showed a 96.6% success rate in preventing painful sickle cell crises, the treatment has now been given the green light.
Around 17,000 people in England live with sickle cell disease, with approximately 4,000 considered eligible for the new therapy. The NHS expects to treat 50 patients per year, focusing on those with severe cases who lack a suitable stem cell donor.
The condition, which primarily affects individuals of African and Caribbean descent, causes red blood cells to become crescent-shaped, obstructing blood flow and leading to severe pain and complications. Patients often face hospital admissions and a significantly reduced life expectancy.
Toby Bakare, a sickle cell patient from London who previously underwent a stem cell transplant, welcomed the approval. “It’s life-changing,” he said. “I was lucky to have a donor, but for those without one, this new therapy is a game-changer.”
Despite the excitement, some patients remain cautious. Mehmet Tunc Onur Sanli, who was diagnosed with sickle cell disease at 11, said: “The idea of no longer needing regular transfusions is incredible, but I want to understand the risks before making a decision.”
Healthcare leaders have hailed the approval as a milestone in addressing health inequalities. Prof. Bola Owolabi, Director of the NHS Healthcare Inequalities Improvement Programme, emphasized the significance of the treatment: “This is a monumental step forward, offering real hope for a brighter future.”
The Sickle Cell Society and other advocacy groups have also praised the move, calling it a turning point in the fight against the disease. Yasmin Sheikh, of the charity Anthony Nolan, described the decision as “a leap forward in treating this life-threatening condition.”
The NHS will now work on rolling out the treatment, ensuring access for those who need it most.
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