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A groundbreaking gene therapy trial has transformed the life of a young boy suffering from an ultra-rare immune disease that had once posed a “death sentence”. Eisa, hailing from Reading, now leads a normal life, playing football and attending school – a reality his family had never imagined before the treatment. The four-year-old was diagnosed with a severe form of leukocyte adhesion deficiency 1 (LAD-1), an inherited disorder that severely impairs the immune system’s ability to combat infections. Eisa’s father, Safdar, described his son as “cuddly and friendly”, expressing gratitude that Eisa’s condition has vastly improved following his participation in the trial.
Eisa’s journey began amidst the ongoing pandemic, with his parents noticing early on that something was amiss. Mr. Safdar recalled his son not appearing well, prompting visits to three different hospitals, ultimately leading them to Great Ormond Street Hospital (GOSH). Upon learning that a bone marrow match was unattainable, GOSH proposed gene therapy as the sole viable option. Mr. Safdar wasted no time in consenting to the treatment, reflecting on how it has empowered Eisa to effectively combat infections independently. He credited GOSH with saving Eisa’s life, emphasizing that his son’s present quality of life would not have been possible without their intervention.
The significance of Eisa’s case laid in the intricate health challenges he faced – a heart complication necessitating surgery compounded by LAD-1’s hindrance to wound healing. Gene therapy emerged as the essential precursor to Eisa’s operation, cultivating hope for his medical suitability. This innovative therapy modulates the patient’s cells, instigating the synthesis of the absent protein crucial for infection resistance. Safdar marveled at Eisa’s progress, acknowledging that his current state exceeds all expectations, with hopes for further advancements like developing speech. Professor Claire Booth from GOSH highlighted the trial’s breakthrough as a gateway to introducing gene therapy across diverse health conditions, encompassing cancer and muscular dystrophy
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