A major phase 3 clinical trial published in the New England Journal of Medicine suggests that blinatumomab, a targeted cancer drug, could replace two cycles of intensive chemotherapy in children with acute lymphoblastic leukaemia.
The findings come from the AIEOP-BFM ALL 2017 trial, a large-scale study examining whether blinatumomab — a drug that directs the immune system to attack cancer cells — can do the work currently done by conventional chemotherapy in paediatric acute lymphoblastic leukaemia (ALL), the most common childhood cancer. The New England Journal of Medicine published both the original research and an accompanying editorial describing blinatumomab as a potential new standard of care in ALL treatment.
Acute lymphoblastic leukaemia is a cancer of the blood and bone marrow that progresses quickly. It affects white blood cells called lymphocytes, and while survival rates in children have improved considerably over decades of chemotherapy-based treatment, that treatment carries serious side effects — including infection risk, organ damage, and long-term developmental effects.
Blinatumomab works differently. It is a type of drug called a bispecific T-cell engager (BiTE), meaning it binds to both cancer cells and immune cells simultaneously, bringing them together so the immune system can destroy the tumour. It is already licensed for use in some adult leukaemia cases, but the AIEOP-BFM ALL 2017 trial tested it specifically in a paediatric setting at phase 3 — the stage of clinical research that directly compares a new treatment against the existing standard.
The New England Journal of Medicine editorial describes the results as marking a shift in how ALL might be treated in children. But phase 3 publication does not mean immediate availability. Regulatory review and NHS approval processes would need to follow before any such treatment could be offered routinely in the UK.
Parents of children currently receiving treatment for ALL should speak to their child’s specialist team about what current and emerging options mean for their situation. NHS 111 can help direct families to the right service if they have concerns and cannot reach their GP.
Source: @NEJM