A thalassaemia patient in Abu Dhabi has become transfusion-independent following gene-therapy treatment, marking a medical milestone in the UAE.
The 43-year-old patient, known only as Fahad, spent years managing transfusion-dependent thalassaemia, requiring treatment every three to four weeks to maintain stable haemoglobin levels.
The condition also led to an overload or iron and a constant need for iron chelation therapy, affecting his quality of life, state news agency Wam reported on Tuesday.
Thalassaemia is a group of blood disorders, sufferers of which lack haemoglobin, the iron-containing protein that carries oxygen in the blood. Low levels can make patients anaemic, and cause fatigue and shortness of breath.
About 18 months ago, Fahad was referred to Yas Clinic and the Abu Dhabi Stem Cells Centre (ADSCC), where scientists were working to introduce advanced gene therapy treatment to the country.
Gene therapy has emerged as one of the most important developments in treating inherited blood disorders, offering a long-term solution for patients without access to a suitable bone marrow donor.
Fahad was identified as a suitable candidate. His treatment involved the collection of his own stem cells, which were genetically modified before being reinfused following preparatory therapy. He has since remained under medical supervision and is no longer reliant on regular transfusion.
Dr Maysoon Al Karam, chief medical officer at Yas Clinic, said the case reflected the UAE's commitment to making advanced therapies accessible to patients domestically. “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 said.
Dr Mohammed Abu Haleeqa, haematologist at Yas Clinic, described the outcome as life-changing for patients who have spent most of their lives dependent on regular transfusion.
“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 said.