Gene therapy allows man to live normal life after severe immune deficiency

Rhys Evans received a pioneering gene therapy at Great Ormond Street Hospital in London after being born with x-linked severe combined immunodeficiency, a condition that left him without a functioning immune system.

As a baby, Evans lived in a sterile bubble to protect him from life-threatening infections. Without the treatment, he faced a future of isolation similar to that of David Vetter, a man who lived in a plastic chamber until his death at age 12.

Professor Adrian Thrasher and a team of experts developed the therapy over 15 to 20 years in a laboratory. The procedure involved taking 100ml of Evans' bone marrow, inserting a new gene into it, and injecting the marrow back into his body. This allowed his own bone marrow to regenerate an immune system. Evans proved the treatment worked when he survived the flu shortly after his second birthday.

Evans now works as a stage crew member at Butlin's in Minehead, Somerset. He has overcome Covid twice and can now attend school, university, and see friends. Since Evans became the first patient to receive the treatment in 2001, more than 130 people have received the therapy. Great Ormond Street estimates that the condition affecting Evans affects about 14 babies a year in England.