GlaxoSmithKline to provide unique child gene therapy

The world's first gene therapy treatment for children to be given regulatory approval has been authorised for sale by a British firm.

GlaxoSmithKline (GSK) was given permission by the European Commission to provide Strimvelis - the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency).

The condition affects the immune system and means children 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.

They will receive the treatment at the Ospedale San Raffaele in Milan.

Martin Andrews, head of GSK's rare disease unit, said: "Today's approval is the result of many years' work with our collaborators in Milan and is the next step towards bringing life-changing treatment to patients with ADA-SCID and their families.

"This is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future."

ADA-SCID is caused by inheriting a faulty gene from both parents and because the children cannot fight infections, they develop severe and life-threatening illnesses.

Without treatment, it is usually fatal within the first year of a sufferer's life.

In the Strimvelis trial, there was a 100% survival rate at three years post-treatment.

Professor Alessandro Aiuti, clinical research coordinator at San Raffaele Telethon Institute for Gene Therapy, said: "This innovative, individualised treatment approach uses a patient's own gene modified stem cells to correct the root cause of the disease.

"It has been gratifying for all of us to see patients affected by this severe immune deficiency growing over the years, being able to play with other children and going to school."