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A 16-year-old girl’s life has been dramatically improved through cutting-edge gene editing that reversed the effects of a severe blood disorder. Vian, born with thalassemia—a condition that impairs haemoglobin, the protein crucial for oxygen transport in red blood cells—had suffered from symptoms including shortness of breath and loss of appetite. For most of her childhood, she depended on monthly blood transfusions, but thanks to the innovative treatment that involved editing her own stem cells, she no longer requires these transfusions.
The gene editing therapy, available to young patients afflicted with either thalassemia or sickle cell disease, is currently offered at three children’s hospitals across the country. Vian, who lives in Coventry, was among the earliest to receive this breakthrough treatment. After undergoing chemotherapy, her own genetically modified stem cells were reintroduced to her body last December. Since then, she has made a strong recovery and described her new life as “so much different” compared to before, explaining that she now has ample energy to engage in activities that were once impossible.
Vian has since enrolled in college, studying health and social care with aspirations to become a nurse—a path inspired by the extensive time she spent in medical environments during her illness. She shared how her improved health has expanded her horizons, allowing her to participate in things other children can do, including physical education, which she used to have to avoid due to her condition. “Before, I couldn’t do most things other kids could,” she said. “I usually sat out of PE. I really love PE, but I couldn’t do that.”
Consultant haematologist Dr Sarah Lawson explained that while the treatment does not eradicate the disease entirely, it functions as a “functional cure” by removing the problematic symptoms. She described the procedure: “We collect the stem cells. They are sent off to a manufacturing laboratory where they are edited… and that changes how those stem cells work and it makes them produce more foetal or baby haemoglobin.” This increase in foetal haemoglobin allows patients with thalassemia to become free from transfusions and helps those with sickle cell disease avoid complications caused by sickle haemoglobin. Dr Lawson reflected on how remarkable it is that a concept of gene therapy she learned about in medical school decades ago has become a reality today.
Meanwhile, 14-year-old JoelSamuel from Oldbury is beginning the same treatment journey to combat the painful effects of sickle cell disease, which often result in hospital stays. His stem cells have recently been collected for gene editing at Birmingham Children’s Hospital and, after chemotherapy expected in six months, his modified cells will be reintroduced. JoelSamuel desires to be able to enjoy playing sports, which currently make him ill due to exhaustion and pain. Though initially apprehensive about the treatment, he expressed hope: “I think it’s good to make me better, so I can go and do stuff that takes my energy.”
JoelSamuel’s parents, Alfred and Juliet, remain optimistic about the therapy’s outcomes. Alfred commented on the limited number of patients treated so far, mostly those with thalassemia, saying, “We are hoping that we might get the same effects from it. Hopefully he might be able to play football after all.” Juliet shared her anticipation for her son’s improved quality of life, saying, “I just can’t wait to see him doing the normal things that everybody does at his age.”
Having completed her own treatment and been discharged from hospital, Vian offered words of encouragement to JoelSamuel as he starts his process: “Imagine all the things you can do while being healthy again. I think you can do it, I did it and so can you. I know it’s going to be really hard and the trauma and everything, the nurses, the medicine, everything, but it’s worth it.”
Read the full article from The BBC here: Read More
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