Cancer

Thanks to advances in medical research and treatment, survival rates for childhood leukaemia have improved dramatically over the past 30 years. However, in around 10-20 per cent of children who have achieved complete remission after initial treatment, leukaemia can come back. The current standard of care for relapsed childhood leukaemia often relies on receiving a bone marrow transplant from a matched donor. These are complex treatments that can carry a significant risk of serious complications.

Recent breakthroughs have shown the exciting potential of gene therapy, which holds promise as an alternative for treating relapsed childhood leukaemia. These include the generation of tumour-targeting ‘killer’ cells, either from a patient’s own blood, or ‘universal’ cells from an unrelated healthy donor, which are modified using genome editing to be ‘invisible’ to the recipient – enabling them to resist rejection.

CAR – chimeric antigen receptor – T-cell therapy works by genetically engineering a patient’s white blood cells (called T-cells) to recognise and destroy cancer cells.

Dr Christos Georgiadis and colleagues from the UCL Great Ormond Street Institute of Child Health have developed such ‘universal’ CAR T-cells with the aid of gene-editing ‘CRISPR’ technology. They are now evaluating the safety and potency of these cells in a Phase 1 clinical trial at Great Ormond Street Hospital.

Although early trial data has been encouraging, reports from similar studies have brought to light instances of disease re-emergence, whereby the cancer cells can evade the CAR therapy by ‘hiding’ the surface markers that researchers are targeting.

Dr Georgiadis’ new research, evaluated initially in mice, will explore whether other markers can be targeted in this way, which could help to strengthen existing CAR therapies for childhood leukaemia.