LONDON: Dr Sher Bahadur Anjum, a British Pakistani paediatric consultant specialising in haematology at Newham Hospital in East London, has successfully overseen the treatment of two young boys with thalassaemia using cutting-edge gene therapy – marking a medical first in the UK.
Thalassaemia is a genetic blood disorder that causes severe anaemia and requires regular blood transfusions for survival. The two boys, aged 11 and 13, both of Bangladeshi heritage, had lived their entire lives dependent on monthly transfusions and frequent hospital visits.
Dr Anjum, in partnership with Great Ormond Street Hospital (GOSH), identified the boys as eligible for a new gene therapy programme. However, it took nearly a year of counselling, reassurance, and building trust before their families agreed to proceed, given the complexity and risks of the new treatment.
After three months of pre-treatment care at Newham Hospital and a four-week stay at GOSH for the gene therapy itself, the boys responded successfully. The therapy involved modifying the patients’ own stem cells to produce healthy haemoglobin, aiming to eliminate the need for future transfusions.
Six months after the treatment, both boys are now living full, healthy lives without any need for blood transfusions. Their families, once burdened by the constant cycle of hospital care, have been given a new lease on life. They are now urging other South Asian families to consider this treatment if eligible.
Dr Anjum called the outcome “extraordinary” and emphasised how this success story demonstrates the transformative potential of gene therapy, as well as the importance of patient-centred, collaborative care within the NHS.