Liver Cancer in Children: CAR T-Cell Therapy Wipes Out Tumour in Trial Case

The New England Journal of Medicine has published correspondence describing a case in which hepatoblastoma (a cancer of the liver that primarily affects young children) went into complete regression after treatment with CAR T-cell therapy. The therapy used T-cells — immune cells taken from the body — that were engineered to carry two additional proteins, interleukin-15 and interleukin-21, designed to boost the cells’ ability to find and destroy cancer.

CAR T-cell therapy works by modifying a patient’s own immune cells in a laboratory so they can recognise and attack specific cancer cells. The addition of interleukin-15 and interleukin-21 — both naturally occurring proteins involved in immune response — is the element that makes this particular approach distinct from existing CAR T-cell treatments already used in some blood cancers.

Hepatoblastoma is rare. It most commonly affects children under five and, in cases where it has spread or returned after chemotherapy, treatment options are limited. That’s why correspondence of this kind, published in one of the world’s leading medical journals, draws attention from oncology researchers.

The findings appear as correspondence rather than a full clinical trial, which means they describe a single case or a small series rather than large-scale tested results. According to the journal’s classification, the piece sits under oncology and gastroenterology.

Research at this stage does not translate into available treatment. Any family affected by hepatoblastoma should speak to their child’s specialist team about current options through the NHS.

For general health concerns, NHS 111 is available online or by phone. Families dealing with a child’s serious illness can also contact CLIC Sargent, the children’s cancer charity, on 0300 123 1801.