A young boy, among the first in the UK to receive a revolutionary £1.65 million gene-editing treatment, has returned to the hospital where he was cared for to express his gratitude to the nurse who helped grant him new-found freedom.
Adam Rehman underwent a one-time gene therapy, Casgevy (also known as exa-cel), designed to cure the debilitating blood disorder thalassaemia.
This pioneering treatment was the first to be licensed using the Crispr gene-editing tool, a scientific breakthrough that earned its inventors the Nobel Prize for chemistry in 2020. The therapy functions by editing faulty genes within a patient’s own stem cells.
Adam, from Oldham, was one of the initial children to benefit from the treatment, according to NHS Blood and Transplant (NHSBT).
While the official price stands at £1.65 million per patient, a confidential agreement between the NHS and manufacturer Vertex suggests the actual cost is likely lower.
The 13-year-old was born with beta-thalassaemia, an inherited blood disorder that impairs haemoglobin function, affecting oxygen transport throughout the body. Symptoms include anaemia and chronic pain, with severe cases posing a life-threatening risk.
Since he was eight months old, Adam relied on monthly blood transfusions. His older sister, who shared the same condition, received a stem cell transplant in 2023, but no suitable stem cell match could be found for Adam.
In November 2024, when he was 12, Adam received the specialist treatment. This involved collecting his stem cells, transporting them to a laboratory for Crispr technology application, and then infusing the edited cells back into his body, prompting the production of healthy red blood cells.
Casgevy offers a vital option for patients who are suitable for a stem cell transplant but lack a donor.
Adam recently reunited with Clair Baron, an NHSBT therapeutic apheresis specialist nurse, who cared for him during his treatment at Royal Manchester Children’s Hospital.
"It feels nice to be back here because I can see the nurses who treated me in the past," Adam said.
"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."
Adam’s father, Anzaq Rehman, 43, from Oldham, added: "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."
Ms Baron expressed her delight: "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 (TAS) at NHS Blood and Transplant, commented: "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."
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