Doctors in London’s Great Ormond Street Hospital (GOSH) are testing a new cancer drug for children as an alternative to chemotherapy. The drug is licensed to treat adults and is now being administered to children with B-cell acute lymphoblastic leukaemia (B-ALL). The doctors report the drug could help around 40 children and young people each year.
‘Blinatumomab or blina is a gentler, kinder treatment,’ said Dr Ajay Vora, consultant paediatric haematologist at GOSH. It is an immunotherapy that precisely targets cancer cells, so the body's own immune system can recognise and destroy them.
Unlike blina, ‘chemotherapies are poisons that kill the leukaemic cells but also kill and damage normal cells - and that is what causes their side effects,’ said Dr Vora.
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Blina comes in a bag of liquid administered through a thin plastic tube that remains running into a vein in the patient's arm for many months. A battery-operated pump controls how quickly the drug trickles into the bloodstream. The kit can be carried in a backpack smaller than an A4 textbook, making it fully portable.
Arthur, 11, was one of the first children to try the drug for his blood cancer at GOSH. He initially underwent traditional chemotherapy, but his family reported it did not work, and left him frail and weak.
The blina administered through a backpack meant that Arthur could do other things, like play on the swings in his local park and sleep in his own bed at night while the treatment was ongoing. In April 2023, the tube was removed from his arm and since then Arthur has been cancer free.
With about 450 children in the UK diagnosed with different types of blood cancers, blina offers hope to both doctors and parents. ‘We believe that blina can replace big chunks of chemo, perhaps up to 80% of it,’ said Dr Vora.
Another targeted immunotherapy drug, chimeric antigen receptor T-cell therapy (CAR-T), has also recently become available. However, it is more expensive than blina and the patient's own cells need to be used and altered, before being given back as the medicine. Further research is needed to evaluate its effectiveness.