Boy born with no immune system thriving thanks to experimental stem cell gene therapy

Credit: PA/GOSH

By Claudia Tanner and Bairbre Holmes, PA

When he was just three weeks old, Andy Cash was diagnosed with a rare life-threatening condition that’s usually fatal by the age of two if left untreated.

Having adenosine deaminase deficient severe combined immunodeficiency (ADA-SCID), his future looked bleak: day-to-day activities like going to school or playing with friends can lead to a dangerous infection.

But thanks to an experimental stem cell gene therapy developed by an international team of researchers including Great Ormond Street Hospital (GOSH), Andy is now a happy, thriving nine-year-old living life to the full.

He is one of 59 of 62 children with ADA-SCID who have seen their immune function restored with no serious complications from the treatment.

The study, published in New England Journal of Medicine, reported on the long-term outcomes for children treated with the gene therapy between 2012 and 2019.

The disease is caused by mutations in the gene that creates an enzyme essential to the immune system.