American and British doctors manage to cure rare and deadly disease of immune system in children

An experimental gene therapy developed jointly by American and British scientists has successfully treated dozens of children with a rare and fatal immune disorder, Euronews writes.

The study included 62 children with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) – a disorder caused by genetic mutations that leave children without working immune systems, making them extremely vulnerable to infections.

Existing therapies for ADA-SCID are expensive and risky, but without treatment it is often fatal within the first two years of life.

The new treatment involves collecting a child’s blood stem cells – which create immune cells – and inserting a virus that delivers a healthy copy of the ADA gene. The corrected cells are then infused back into the patient, allowing them to produce healthy immune cells.

After the procedure, it takes six to 12 months for the immune system to reach normal levels, the researchers said.

Fifty-nine patients were successfully treated with the gene therapy between 2012 and 2019. Since then, their immune functions have remained stable and they have not reported serious complications.

Five of the children are still healthy more than a decade later.

The researchers said the study is the largest and longest follow-up of a gene therapy of this kind.