Gene Therapy Breakthrough in Abu Dhabi Frees Thalassaemia Patient from Lifelong Transfusions

A significant medical milestone has been achieved in the UAE as a thalassaemia patient in Abu Dhabi has successfully become independent of blood transfusions following advanced gene therapy treatment. The 43-year-old patient, identified as Fahad, had for years managed transfusion-dependent thalassaemia, a condition necessitating treatment every three to four weeks to maintain adequate haemoglobin levels. This chronic condition also resulted in iron overload and a continuous requirement for iron chelation therapy, significantly impacting his quality of life, according to state news agency Wam.

Thalassaemia is a group of inherited blood disorders characterized by a lack of haemoglobin, the protein responsible for carrying oxygen in the blood. Insufficient haemoglobin levels can lead to anaemia, causing symptoms such as fatigue and shortness of breath.

Approximately 18 months ago, Fahad was referred to Yas Clinic and the Abu Dhabi Stem Cells Centre (ADSCC), where researchers were pioneering the introduction of advanced gene therapy to the country. Gene therapy is emerging as a pivotal development in treating inherited blood disorders, offering a potential long-term solution for patients who do not have access to a compatible bone marrow donor.

Fahad was deemed a suitable candidate for the treatment. His therapy involved the extraction of his own stem cells, which were then genetically modified. These modified cells were subsequently reinfused into his body after preparatory treatment. Since the procedure, Fahad has remained under ongoing medical supervision and is no longer dependent on regular transfusions.

Dr. Maysoon Al Karam, chief medical officer at Yas Clinic, highlighted that this case underscores the UAE's dedication to making cutting-edge therapies accessible to patients within the country. "This marks an important milestone in expanding access to innovative treatment options and reinforces Abu Dhabi's position as a regional centre for advanced health care and medical innovation," she stated.

Dr. Mohammed Abu Haleeqa, a haematologist at Yas Clinic, described the outcome as life-altering for individuals who have spent the majority of their lives reliant on frequent blood transfusions. "Gene therapy offers the opportunity to address the underlying cause of the disease using the patient's own genetically modified cells, providing an advanced treatment option for patients who may not have suitable donors for transplantation," Dr. Abu Haleeqa explained.