Rhys Evans, the first infant in the UK to receive gene therapy, recently returned to Great Ormond Street Hospital to thank the medical team that saved him. Diagnosed with Severe Combined Immunodeficiency 25 years ago, Evans survived a prognosis that gave him only two years to live.
The 'bubble' and the two-year life expectancy of Rhys Evans
Rhys Evans was born in Cardiff and faced a precarious start to life due to Severe Combined Immunodeficiency, a rare genetic condition that left him without a functioning immune system. According to the report, the situation became critical in 2001 when Evans fell ill after his mother, Marie, stopped breastfeeding, leading to a series of dangerous infections. Because doctors in Wales were unable to provide a diagnosis, the infant was flown by helicopter to London for specialist care.
Upon arriving at Great Ormond Street Hospital (GOSH), Rhys Evans was placed in a sterilized 'bubble' to prevent any contact with external pathogens. Every item that entered his environment—including food, nappies, and toys—had to be rigorously sterilized. At the time , medical experts provided a grim outlook, estimating that the child had a life expectancy of only two years without an intervention that could rebuild his immune defenses.
Kimberly Gilmour and the risks of the 2001 experimental trial
The treatment that saved Rhys Evans was an experimental gene therapy designed to replace his faulty genes, a procedure that had never been attempted in the UK at that time.. Kimberly Gilmour, who processed the cells for the procedure in 2001 and now serves as the chief of laboratory medicine at Great Ormond Street Hospital, noted that the process was daunting because the team had never worked with fresh cells before.
As the report says, the tension of the moment was palpable, with Rhys Evans' parents, Mark and Marie, watching the laboratory team through a glass partition. The medical staff admitted they did not know if the experimental treatment would actually result in a cure. However, within weeks of the procedure, the body of Rhys Evans began to develop its own immune system, marking a historic success for British medicine.
From one patient to 135 children and 35 different therapies
The success of the trial involving Rhys Evans served as a catalyst for a broader revolution in genomic medicine. Great Ormond Street Hospital has since transitioned from a site of experimental firsts to a global center of excellence, treating more patients with gene therapy technology than any other institution in Europe. this trajectory reflects a wider shift in medicine where once-untreatable genetic disorders are now targeted at the molecular level.
Since that first breakthrough 25 years ago, the hospital has delivered 35 different cell and gene therapies to more than 135 children suffering from various rare conditions. The evolution of the GOSH program demonstrates how a single high-risk success can build the clinical confidence and infrastructure necessary to scale life-saving treatments for hundreds of other families facing rare diseases.
The 5% gap and the ongoing need for antibiotics
Despite his success, the recovery of Rhys Evans is not absolute. Now 25 years old and working at Butlins and a coffee shop in Minehead, Evans describes himself as being "95 per cent normal." A critical detail mentioned in the report is that he must continue to take antibiotics to maintain his immune system's functionality, suggesting that the 2001 therapy provided a lifeline rather than a total biological reset.
This leaves several specific medical questions unanswered: it remains unclear what the long-term stability of this first-generation gene therapy looks like as Evans enters adulthood, and the report does not specify the exact dosage or frequency of the antibiotics required to keep him healthy. Furthermore, while the hospital celebrates the 135 children treated, the source does not provide the current survival or health rates for the other patients who underwent these 35 different therapies, leaving the broader success rate of the GOSH program partially obscured.
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