Glaxo’s £500k gene drug triumph

A BREAKTHROUGH gene therapy designed by Glaxo Smith Kline to treat ‘bubble baby syndrome’ has been backed by UK healthcare regulators.

Strimvelis is the most expensive medicine ever approved by the National Institute for Health and Care Excellence (Nice), costing about £525,000.

It was developed by GSK to treat ADA-SCID, an extremely rare, inherited condition that leaves children without a properly functioning immune system.

About three babies are born in England with ADA-SCID every year. It means they are at high risk of infection and so must usually live in isolation.

Sufferers were dubbed ‘bubble babies’ following a 1976 film The Boy in a Plastic Bubble, starring John Travolta, which depicted the condition.

Previously, it could only be treated with risky stem cell transplants, for which it can be difficult to find matching donors. But GSK’s new medicine means more children will be able to get help.

Nice said that it could be lifechanging, allowing youngsters afflicted with the condition to go to school and socialise with friends, without fear of catching deadly infections.

The treatment involves removing the patient’s bone marrow cells, modifying them and then returning them via drip.

Currently it can only be administered at a hospital in Milan, which means patients have to travel to Italy.

Professor Carole Longson, of Nice, added: ‘ Strimvelis represents an important development, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments.

‘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.’

Under the new draft guidance which has been published by Nice, the GSK treatment will be recommended when no matched stem cell donor is available.