A revolutionary gene editing treatment for sickle cell disease, Casgevy (exa-cel), has received approval from the National Institute for Health and Care Excellence (Nice) for use on the NHS. This groundbreaking development offers a potential cure for severely affected patients, marking a significant step forward in treating this debilitating and painful condition.
A groundbreaking £1.65 million treatment has been approved for use on the NHS , offering patients with severe sickle cell disease a potential cure. The National Institute for Health and Care Excellence (Nice) has approved Casgevy , also known as exa-cel, for some patients with the genetic condition. It is estimated that around 1,700 people could be eligible for the treatment, with approximately 50 patients receiving it each year.
Professor Bob Klaber, director of strategy, research and innovation at Imperial College Healthcare NHS Trust, which led the UK arm of clinical trials for exa-cel, expressed his pride in being part of this groundbreaking research and international collaboration. He highlighted the treatment as a true medical innovation, offering patients with no other options a potential cure for the painful and debilitating symptoms of their disease. He also noted its promising potential for research avenues in other genetic diseases.For sickle cell patients like Lanre Ogundimu, the drug could be a game-changer. In 2018, she suffered a stroke, a pulmonary embolism, and a blood transfusion reaction due to the disorder. This resulted in a 30-day hospital stay, six months of physiotherapy, over 12 months of therapy, and nine months off work. She described feeling weak all the time, stating that this was the lowest point in her life. It impacted her freedom and independence, her income, her career trajectory, and her ability to contribute to society. Another patient, Mehmet Tunc Onur Sanli, 42, from London, was diagnosed with the disorder at age 11. He underwent surgery on his spleen at six and a hip replacement at 22. He suffers from regular sickle cell crises, experiencing severe pain that necessitates frequent hospital visits. He expressed that while there are still aspects to consider with the new treatment, the possibility of not having to go to the hospital regularly would be a dream come true.Casgevy, which earned its inventors the Nobel Prize for Chemistry in 2020, works by editing the faulty gene in a patient's own stem cells. It serves as an option for patients when a stem cell transplant is suitable but no donor can be found. The treatment was initially rejected by Nice in March last year due to the need for further details regarding its effectiveness and a commercial agreement for the drug, which has a list price of £1.65 million. The confidential agreement reached with the drug company, Vertex, keeps the NHS's exact payment amount undisclosed. Clinical trials demonstrated that all patients who received exa-cel avoided hospital admission for a year following treatment, with almost 98% still avoiding hospitalization around 3.5 years later. The treatment will be offered at specialist NHS centers in London, Manchester, and Birmingham.Amanda Pritchard, NHS England chief executive, stated that the NHS would be funding the new treatment immediately. She emphasized its potential to be transformative for people with sickle cell disease, which can be extremely debilitating and painful. She believes it offers hope of a cure for patients facing the severe form of the disease, potentially enabling them to live free from the fear of sickle cell crises. University student Funmi Dasaolu, who has experienced chronic fatigue and pain throughout her life, expressed her joy over the approval of exa-cel after months of campaigning. The 31-year-old from Oxfordshire, who has been hospitalized numerous times due to the condition, including seven hospitalizations in 2022 and regular blood transfusions for the past five years, sees this approval as truly transformative, offering the chance of a life free from the disease. She envisions a future where she can grow old, fulfill her dreams, and live a pain-free life
Sickle Cell Disease NHS Treatment Cure Gene Editing Casgevy Exa-Cel Nobel Prize
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