Gene therapy opens new possibilities for thalassemia patients in UAE

Yas Clinic, in collaboration with Abu Dhabi Stem Cells Center (ADSCC), has achieved promising results in the treatment of thalassemia through advanced gene therapy, enabling Fahad, a 43-year-old patient, to become independent from regular blood transfusions after years of relying on them to manage his condition. 

For many years, Fahad lived with transfusion-dependent thalassemia, requiring blood transfusions every three to four weeks to maintain stable hemoglobin levels. In addition to the ongoing burden of treatment, he experienced a range of complications associated with long-term transfusions, including iron overload and the need for continuous iron chelation therapy, which impacted his overall health and quality of life. 

Approximately a year and a half ago, Fahad was referred to Yas Clinic and ADSCC at a time when both institutions were working to introduce advanced gene therapy treatments to the UAE. Gene therapy represents one of the most significant advancements in the treatment of inherited blood disorders, offering a potential long-term solution for patients who may not have access to a suitable donor for bone marrow transplantation. 

Following comprehensive medical evaluations, extensive testing, and detailed multidisciplinary assessments, Fahad was identified as a suitable candidate for gene therapy. His treatment journey coincided with national efforts to make this advanced therapy available within the UAE, supported by the Department of Health and the country's leadership, creating new opportunities for patients to access innovative treatments closer to home. 

The treatment process involved collecting Fahad's own stem cells, which were subsequently genetically modified before being reinfused following preparatory therapy. The approach, which shares similarities with an autologous transplant, is designed to address the underlying cause of the disease using the patient's own genetically modified cells. 

Fahad underwent gene therapy last year and has since remained under close follow-up with the multidisciplinary medical team. Today, he is transfusion independent and continues to enjoy improved health and greater freedom from the frequent hospital visits that had been a defining part of his life for many years. 

Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, said: "Gene therapy represents a transformative advancement in the treatment of inherited blood disorders and reflects the UAE's commitment to making the most advanced therapies available to patients. The success of this case marks an important milestone in expanding access to innovative treatment options and reinforces Abu Dhabi's position as a regional center for advanced healthcare and medical innovation." 

She added: 

"We remain committed to investing in medical innovation and expanding access to advanced therapies that improve patient outcomes and quality of life while ensuring patients can receive world-class care within the UAE." 

Dr. Mohammed Abu Haleeqa, Hematologist at Yas Clinic, said: "For patients who have spent most of their lives dependent on regular blood transfusions, reaching a stage where transfusions are no longer required can be truly life-changing. 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." 

He added: "Seeing patients regain their independence from transfusions and return to their daily lives with greater confidence and stability is one of the most rewarding outcomes of this therapeutic advancement." 

This achievement represents another important milestone in the development of gene therapy services within the UAE and reflects the ongoing commitment of Yas Clinic and ADSCC to providing patients with access to the latest treatment innovations for inherited blood disorders.