Funmi Dasaolu, a determined 31-year-old university student from Oxfordshire, is among those who can now look forward to a future of hope, thanks to a revolutionary new gene therapy treatment. This treatment, recently approved for NHS use, promises to transform the lives of patients suffering from severe sickle cell disease, offering the possibility of a life without the chronic pain and debilitating effects of the condition.
The treatment, known as exacel (formerly Casgevy), utilizes the groundbreaking gene-editing technology called CRISPR. This technique, which earned its inventors the Nobel Prize for Chemistry in 2020, works by editing the faulty gene in a patient’s own stem cells. By making this correction, the treatment helps address the root cause of sickle cell disease, potentially offering long-lasting relief. NHS officials estimate that around 50 people per year will benefit from this treatment, marking a major step forward in the fight against this life-limiting condition.
For Dasaolu, who has faced severe health challenges due to sickle cell disease throughout her life, this approval represents a significant milestone. In 2022 alone, she was hospitalized seven times due to complications related to the disease. She has also endured chronic fatigue and intense pain, and for the past five years, regular blood transfusions have been her only means of symptom management. Now, with exacel offering new hope, Dasaolu feels a sense of optimism that has been previously out of reach.
In a heartfelt statement, she expressed, “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 fulfill our dreams, and to live a pain-free life.”
Sickle cell disease is a group of inherited conditions that affect the red blood cells, causing them to become abnormally shaped. These sickle-shaped cells do not survive as long as healthy red blood cells and can block blood vessels, leading to painful episodes and serious complications. The approval of exa-cel marks a potential breakthrough, giving many individuals a chance to lead a more normal life.
Another patient, Toby Bakare, a 35-year-old TV producer from South London, also welcomed the approval of the new treatment. As a child, Bakare frequently missed school due to the painful sickle cell crises that would land him in the hospital up to six times a year. He later underwent a stem cell transplant from one of his siblings, but for many others, exa-cel will offer a new opportunity for relief.
Bakare shared his excitement, stating, “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.”
For many patients, like Lanre Ogundimu, the impact of sickle cell disease is devastating. In 2018, Ogundimu suffered a stroke, pulmonary embolism, and a severe blood transfusion reaction, all of which landed her in intensive care for weeks. Following this, she endured months of rehabilitation, during which her life was turned upside down. “It impacted my freedom and independence, my income, my career trajectory, and my ability to contribute towards society,” she recalled.
With exa-cel now available, patients like Dasaolu, Bakare, and Ogundimu hold out hope for a brighter future. The approval of this gene therapy is a significant step forward in the medical community’s ongoing battle against sickle cell disease, providing new possibilities for those who have long suffered in silence.
