In a groundbreaking medical achievement, British-Pakistani pediatric consultant Dr. Sher Bahadur Anjum has successfully treated two young thalassemia patients with gene therapy, restoring them to complete health and normality for the first time in the UK. Dr. Anjum, a specialist in hematology at Newham Hospital in East London, part of Barts Health NHS Trust, led a team that treated the two boys, aged 11 and 13, who are of Bangladeshi descent.
Thalassemia is a genetic blood disorder particularly common among people of Pakistani, Indian, and Bangladeshi origin. Individuals with this condition have low iron and oxygen levels, leading to severe anemia, fatigue, and shortness of breath. Before this treatment, the two patients required monthly blood transfusions, which significantly impacted their quality of life. The only known cure is a stem cell or bone marrow transplant, but this is rarely performed due to the associated risks and the difficulty of finding suitable donors.
Thanks to an innovative gene therapy administered by Dr. Anjum and his team, in collaboration with Great Ormond Street Hospital (GOSH), the two boys are now living healthy, happy lives without the need for hospital visits or regular transfusions. The treatment, which lasted four weeks at GOSH, was preceded by three months of extensive pre-gene therapy care at Newham Hospital. This preparation included blood testing, vaccinations, and maintaining correct hemoglobin levels. The boys’ care at Newham continued long after the treatment, with ongoing monitoring and management of any side effects.
When Dr. Anjum first presented the gene therapy option to the families, both were hesitant due to the newness of the treatment and the required lengthy hospital stay. However, after nearly a year of continuous conversations, reassurance, and building trust, the families were convinced and agreed to proceed.
Dr. Anjum, who originally hails from Gilgit Baltistan’s Astoria village and studied at Abbottabad Medical College, 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 hemoglobin, and then return these cells to the body after special preparation. The goal is that your child’s body starts making enough hemoglobin on its own, so they no longer need regular blood transfusions.”
He added that this success is a testament to the families’ courage and the power of gene therapy. “Both of my patients had grown up knowing nothing but hospital visits and transfusions… It took almost a year of continuous conversations, reassurance, and building trust before they agreed,” he said. Now, six months after the treatment, their lives have been transformed. “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 believes this breakthrough offers a beacon of hope for families affected by thalassemia, allowing them to envision a future where their children are free from the shadow of chronic illness. He emphasized that this achievement highlights what is possible through collaboration within the NHS when patient care is the central focus.
