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23.10.17

NICE approves first ever gene therapy for ‘bubble baby syndrome’

The first ever gene therapy on the NHS, designed to treat infants with the very rare immune deficiency condition adenosine deaminase deficiency (ADA-SCID), has been approved for use on the NHS.

The disease has the nickname ‘bubble baby syndrome’ because infants tend to be extremely vulnerable to infection and have to live in isolation to minimise the risk.

Until now the only answer to the condition has been risky stem cell transplant treatment, made even more difficult to complete safely by the lack of good matches.

The new treatment, Strimvelis, is only the second-time gene therapy for an inherited disease has been licensed anywhere in the world. The treatment involves removing bone marrow cells and modifying them outside the body to produce the working ADA enzyme.

Although the syndrome only affects three babies in Britain each year, this is particularly important as it represents the first-time any gene therapy has been approved and funded by the NHS.

Young patients left untreated will die before school age, but the new therapy has been developed as a one-off cure to the condition, meaning children would be able to go to school and socialise without fear of a dangerous infection.

“Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments,” said Professor Carole Longson, director of the centre for health technology assessment at NICE.

“This means that children born with ADA-SCID will now have a better chance of being able to lead as near normal a life as possible, going to school, mixing with friends, free from the constant threat of getting a potentially life-threatening infection.”

Public consultation of the draft guidance from NICE will be open until 13 November.

Top image: Reptile8488

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