LONDON: British-Pakistani paediatric consultant Dr Sher Bahadur Anjum has successfully treated two young thalassaemia patients through gene therapy, transforming their lives to complete normality — for the first time in the UK.
Dr Anjum, who specialises in haematology (blood disorders) at Newham Hospital in East London, part of Barts Health NHS Trust, led a team of carers at the Newham Hospital to treat the two young boys, now aged 11 and 13, of Bangladeshi background.
Thalassemia is a genetic condition that is most common in those who are Pakistani, Indian, and Bangladeshi. It is a blood disorder, and people with thalassemia have low iron and oxygen levels in the body. This can make them severely anaemic — tired, short of breath, and pale.
To treat their thalassemia, the two patients needed to go to the hospital for blood transfusions every month, which had a major impact on their quality of life. The only cure is a stem cell or bone marrow transplant, but this is not done very often because of the risks involved, and it can be difficult to find donors.
Thanks to an innovative new gene therapy administered by Dr Anjum and his team, working with Great Ormond Street Hospital (GOSH), the two young boys, now aged 11 and 13, are now living healthy, happy lives without further hospital visits or regular blood transfusions.
Before getting the 4-week treatment at GOSH, Dr Anjum and the healthcare team at Newham Hospital conducted extensive pre-gene therapy care over three months, which included blood testing, vaccinations and maintaining the correct haemoglobin levels. Their care at Newham also continued long after the treatment, with monitoring, blood counts and managing the side effects.
When Dr Anjum first approached the families, they were both hesitant to start this therapy as it was new, and requires an initial longer hospital stay. However, after almost 12 months of speaking to them, listening and answering their questions and concerns, they were reassured to start the treatment for their children.
Now their lives have changed forever thanks to the treatment, and they want to encourage other South Asian parents to take up the offer if doctors find them eligible.
Dr Anjum, who originally studied in Gilglit Baltistan’s Astoria village and then Abbottabad Medical College (AMC) told Geo News: “Gene therapy is a new treatment that aims to fix the problem at its root. Doctors take some of your child’s own blood stem cells, add a healthy copy of the gene needed to make haemoglobin, and then return these cells to the body after special preparation. The goal is that your child’s body starts making enough haemoglobin on its own, so they no longer need regular blood transfusions. In many children, this has already worked successfully.
“Both of my patients had grown up knowing nothing but hospital visits and transfusions. Their parents had shouldered years of emotional and practical struggles, doing everything possible to keep their children well. When Barts Health NHS Trust was asked to identify patients for the new gene therapy programme, we saw a once-in-a-lifetime opportunity.
“But convincing the families was not simple. Gene therapy is new, complex, and frightening to parents who have already endured so much. Understandably, both families were hesitant and fearful of risks. It took almost a year of continuous conversations, reassurance, and building trust before they agreed. At the Great Ormond Street Hospital, I was given an honorary contract so I could support the children and their families before, during, and after the procedure. The therapy itself was intense, requiring months of preparation, but the courage of these families carried them through.
“Six months on, the transformation is extraordinary. These children, once tethered to hospital beds and transfusion schedules, are now living normal lives without the need for blood transfusions. Their energy has returned, their futures are brighter, and their families have finally been released from the unending cycle of hospital visits.”
Dr Anjum said that for the first time, families affected by thalassaemia can imagine a future where their children are free from the shadow of chronic illness. “It shows the power of gene therapy to turn despair into hope, and it highlights what collaboration within the NHS can achieve when we put patients and families at the heart of care.”
