Teenager Celebrates New Freedom After £1.65 Million Gene-Editing Therapy
A teenage boy from Greater Manchester has spoken of his newfound freedom after becoming one of the first children in the United Kingdom to receive a revolutionary gene-editing treatment valued at £1.65 million. Adam Rehman, now 13, recently returned to the hospital where he underwent the pioneering procedure to personally thank the specialist nurse who played a crucial role in his care.
Pioneering Treatment for Debilitating Condition
Adam, who hails from Oldham, was born with beta-thalassaemia, a serious inherited blood disorder that severely impairs the body's ability to produce functional haemoglobin. This condition disrupts oxygen transport throughout the body, leading to debilitating symptoms including severe anaemia and chronic pain. For patients with severe forms like Adam's, the disorder can be life-threatening without regular medical intervention.
From just eight months old, Adam's survival depended on monthly blood transfusions – a gruelling routine that dominated his childhood and limited his ability to participate in normal activities. His older sister, who shared the same genetic condition, received a stem cell transplant in 2023. However, despite extensive searches, medical teams could not find a suitable stem cell donor match for Adam.
The Breakthrough of Casgevy Therapy
In November 2024, when Adam was 12 years old, he received the groundbreaking treatment known as Casgevy (also called exa-cel). This one-time gene therapy represents a monumental scientific achievement as the first treatment licensed using the Crispr gene-editing tool – technology whose inventors were awarded the Nobel Prize for Chemistry in 2020.
The complex treatment process involved:
- Collecting Adam's own stem cells
- Transporting them to a specialist laboratory
- Using Crispr technology to edit the faulty genes within the cells
- Reinfusing the corrected cells back into his body
These edited cells then began producing healthy red blood cells, effectively curing the underlying genetic cause of his thalassaemia. While the official price tag stands at £1.65 million per patient, NHS Blood and Transplant has confirmed that a confidential agreement with manufacturer Vertex Pharmaceuticals means the actual cost to the health service is likely substantially lower.
Emotional Hospital Reunion
Adam recently made an emotional return to Royal Manchester Children's Hospital where he reunited with Clair Baron, the NHSBT therapeutic apheresis specialist nurse who cared for him throughout his treatment journey. "It feels nice to be back here because I can see the nurses who treated me in the past," Adam said during the visit. "It's really good to see Clair and to be able to say thank you to her – she was the start of my treatment and now I'm much better. But it is nice to have my freedom back and not be in hospital anymore."
His father, Anzaq Rehman, 43, echoed this sentiment, stating: "It feels great to be back here and to be reunited with Clair – we're so grateful for the role that she played in Adam's journey, she was that first step towards where we are now and that's so special."
Transforming Lives Through Medical Innovation
Ms Baron expressed her profound joy at seeing Adam's remarkable progress: "It's absolutely wonderful to see Adam, it genuinely is, seeing how well he is doing is just brilliant. To see him now, out on the other side, back at school and being able to enjoy activities in a way he couldn't before, is just incredible. The gene therapy has given him his life back and it's an honour to be a part of his story."
Teresa Baines, head of therapeutic apheresis services at NHS Blood and Transplant, highlighted the broader significance of such treatments: "It's incredible to see Adam doing so well in such a short amount of time. Gene therapies are truly life-changing treatments. By collecting the cells, our TAS teams are setting patients off on their journey and that's a real privilege. Having Adam visit and hearing how much the treatment has changed his life, really does help to see how important the work that we do is."
This case represents a significant milestone for the NHS and medical science in the United Kingdom, demonstrating how cut-edge genetic therapies can transform the lives of patients with previously incurable conditions. For Adam Rehman, the treatment has meant exchanging a childhood dominated by hospital visits for the simple pleasures of school, play, and the freedom he once could only imagine.
