Boy hails new-found ‘freedom’ after £1.6m treatment
A boy who was one of the first children to be given a £1.65 million treatment has returned to the hospital where he received his care to thank his nurse for his new-found freedom.
Adam Rehman was given a one-time gene editing therapy to cure blood disorder thalassaemia.
Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020.
The treatment works by editing the faulty gene in a patient’s own stem cells.
Adam, from Oldham, was one of the first children to receive the treatment, NHS Blood and Transplant (NHSBT) said.
A confidential deal has been struck between the NHS and manufacturer Vertex, which means it is likely to be paying less than the official price of £1.65 million per patient.
The 13-year-old was born with beta-thalassaemia, an inherited blood disorder that means the haemoglobin in the blood does not work properly, affecting how oxygen is carried around the body.
Symptoms include anaemia and chronic pain, and in severe cases the condition can be life threatening.
Since he was eight months old, Adam relied on monthly blood transfusions.
His older sister, who had the same condition, was given a stem cell transplant in 2023 but no stem cell match could be found for Adam.
In November 2024, when he was 12, Adam received the specialist treatment, which involves his stem cells being collected and taken to a laboratory where the Crispr technology is used.
The edited cells are then infused back, which prompts the body to produce healthy red blood cells.
Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found.
Now Adam has reunited with the nurse that cared for him during treatment – NHSBT therapeutic apheresis specialist nurse Clair Baron.
The meeting took place at Royal Manchester Children’s Hospital, where he had his treatment.
Adam said: “It feels nice to be back here because I can see the nurses who treated me in the past.
“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 said: “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, said: “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.”
Published: by Radio NewsHub