13-year-old boy who received groundbreaking gene therapy to cure thalassaemia is reunited with nurse who started his journey

A 13-year-old boy has been reunited with the nurse who started him on his journey as one of the first children to receive gene therapy to treat beta thalassaemia, in an emotional meeting where the nurse exclaimed that "seeing how well he is doing is just brilliant".

Adam Rehman, from Oldham, visited NHS Blood and Transplant (NHSBT) Therapeutic Apheresis (TAS) Specialist Nurse Clair Baron to update her on his progress and revisit the place where his journey began. He and his parents visited ward 79 at Royal Manchester Children's Hospital, where he spent a week for his stem cell collection last November and a further 5 weeks after his stem cell transplant in June this year.

NHSBT nurse Clair travels to hospitals to carry out specialist therapies. She travelled to the Manchester hospital to treat Adam last year and last month, they met Clair in the very room he spent his time in, with the nurse declaring how wonderful it is to see him now, seven months after he received his cells back, doing so well.

Clair said: "It's absolutely wonderful to see Adam, it genuinely is. Seeing how well he is doing is just brilliant! I remember him during the collection, when he was just about to embark on this big, new journey. He was so brave - he spent the few days of the cell collection mainly watching his iPad and dealing with everything he was going through so incredibly well. So, 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.

"As Therapeutic Apheresis Services nurses, we're often at the start of someone's journey and we don’t get to see how they get on, so it's a real joy to be able to do that. Thank you to Adam and his parents for coming in to see me today – it really has made my day."

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 to be 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.

"When you come back after the treatment is done and you see everyone again, you can look at it from a different point of view. It’s wonderful to see everyone."

Adam, sitting in a hospital bed, holding his collected stem cells Adam was born with beta-thalassaemia, an inherited blood disorder that means the haemoglobin in his blood does not work properly, affecting how oxygen is carried around the body. The disorder can impact greatly on quality of life, causing anaemia and chronic pain amongst other symptoms. In the most severe cases, beta-thalassaemia can become life threatening.

Since being 8-months-old, Adam has relied on monthly blood transfusions to help him feel well. His older sister received a stem cell transplant in 2023 to treat the same condition, however no stem cell donor match could be found for Adam and his medical team decided to hold off and wait for gene therapy to become available.

The therapy was approved in August 2024 and in November, Adam became one of the first paediatric patients to have his stem cells collected for the therapy, which uses gene-editing CRISPR technology to encourage the patient’s stem cells to produce functioning haemoglobin. The cells were then returned to Adam via an infusion.

Statements

Statement from Dr Joseph Sharif

Dr Joseph Sharif, Consultant Haematologist at NHS Blood and Transplant, says:

"The new gene-editing treatment enables patients with thalassaemia to produce healthy red blood cells, removing the need for regular blood transfusions, and the associated hospital visits and medication. This marks a significant milestone in the therapeutic landscape, supporting efforts to improve the lives of our patients.

"It's a joy to see Adam doing so well after the treatment, we wish him all the best."

Statement from Teresa Baines

Teresa Baines, Head of Therapeutic Apheresis Services 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. We wish Adam and his family all the best and look forward to being a part of many more patients' journeys."

How you can help

Find out more about Therapeutic Apheresis Services treatments.

Visit out Give Blood website to book a blood or plasma donation appointment or call 0300 123 23 23 to find your nearest session.

Adam and his family would like to raise awareness of the Transport for Sick Children charity, in Stockport, Greater Manchester, who provided travel for Adam during his treatment