First Gene Therapy for Children is Approved in Europe


Radical treatment for rare ‘bubble boy’ disorder has a 100% survival rate

The European Commission has given approval for British drug firm GlaxoSmithKline to produce the world’s first gene therapy treatment for children. GSK will produce Strimvelis, the first stem cell gene therapy to treat patients with severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID)

The disease is caused by caused by adenosine deaminase deficiency (ADA-SCID). This is when the body doesn’t produce a key enzyme in maintaining the immune system, leading to the build up of toxins. As a result, children affected by the disease are unable to fight off everyday infections. Around 15 children a year in Europe are affected by ADA-SCID and the treatment will help those for whom home and stem cell donor is not available. The treatment works by using stem cells obtained from the child’s own bone marrow. Once the cells are extracted they are tweaked with DNA for a working copy of the enzyme introduced.

These stem cells are then reintroduced to the patient, who should then be able to produce their own working copies of the immune enzyme. Trial data have shown a 100 per cent survival rate for children receiving Strimvelis up to three years after the treatment.

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