A groundbreaking treatment worth £1.65 million has been approved for use on the NHS, offering hope to patients with severe sickle cell disease – a significant milestone in the medical field. The National Institute for Health and Care Excellence (Nice) has given the green light to Casgevy for eligible patients with this genetic condition, potentially impacting around 1,700 individuals. NHS officials anticipate that approximately 50 people each year will benefit from this innovative treatment.
Professor Bob Klaber, the director of strategy, research, and innovation at Imperial College Healthcare NHS Trust, which led the UK arm of the clinical trials for exa-cel, expressed his pride in the groundbreaking research and international academic collaboration that paved the way for this remarkable development. He emphasized the significance of this treatment, offering a potential cure for the painful and debilitating symptoms of sickle cell disease, with promising research avenues opening up for other genetic conditions.
For patients like Lanre Ogundimu, who faced a series of health challenges due to sickle cell disease, this treatment could be a game-changer. Lanre’s struggles included a stroke, pulmonary embolism, and a blood transfusion reaction in 2018, leading to prolonged hospitalization, physiotherapy, and a significant impact on her daily life. She shared the emotional toll of feeling weak and helpless, highlighting the far-reaching consequences of the disease on her freedom, independence, and career aspirations.
Understanding Sickle Cell Disease
Sickle cell disease encompasses a group of inherited conditions affecting red blood cells, with sickle cell anaemia being the most severe form. The disorder causes red blood cells to adopt a sickle shape, leading to premature death and reduced oxygen-carrying capacity in the body. Predominantly found in individuals with an African or Caribbean family background, sickle cell disease arises from a gene that influences red blood cell development. While having the gene doesn’t guarantee the disease, it indicates carriers of the sickle cell trait.
Symptoms of sickle cell disease include painful episodes known as sickle cell crises, heightened susceptibility to infections, and anaemia, resulting in fatigue and breathlessness. At present, stem or bone marrow transplants offer the only cure for sickle cell disease, albeit infrequently due to the associated risks involved.
Mehmet Tunc Onur Sanli, diagnosed with the disorder at a young age, recounted his struggles with regular sickle cell crises, vividly describing the excruciating pain that necessitated numerous hospital visits. Despite acknowledging the considerations surrounding the new treatment, he expressed his longing for a life free from frequent hospital visits, underscoring the transformative potential of this breakthrough.
The Mechanism of the Treatment
Casgevy, also known as exa-cel, received the Nobel Prize for chemistry in 2020 for its innovative approach to treating sickle cell disease. The treatment involves editing the faulty gene in a patient’s stem cells, providing an option when a stem cell transplant is appropriate but a donor is unavailable. Initially declined by Nice due to efficacy concerns, the treatment’s approval required further data and a commercial agreement, considering its list price of £1.65 million.
During clinical trials, patients receiving exa-cel avoided hospitalization for a year post-treatment, with nearly 98% maintaining hospital-free status over 3.5 years. The treatment will be accessible at specialized NHS centers in London, Manchester, and Birmingham, offering a beacon of hope for patients grappling with sickle cell disease.
NHS England chief executive Amanda Pritchard hailed the approval of this new treatment, emphasizing the positive impact it could have on individuals with sickle cell disease. She noted that the treatment could be life-changing for patients facing severe forms of the disease, offering them a chance at a healthier, symptom-free life.
University student Funmi Dasaolu, who has battled chronic fatigue and pain due to sickle cell disorder, welcomed the approval of exa-cel with gratitude and optimism. Recounting her numerous hospitalizations and years of blood transfusions, Funmi expressed her joy at the transformative potential of this treatment, painting a picture of hope, dreams, and a future free from the shackles of the disease.
