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Treatment tackles 'incurable' leukaemia

An experimental drug – originally developed using animal studies – has cleared a UK teenager of her cancer, when standard treatments have failed.

13-year-old Alyssa (pictured), from Leicester, was diagnosed in 2021 with T-cell acute lymphoblastic leukaemia (T-ALL), a cancer in which immune T-cells, which usually help to protect the body, multiply and grow out of control.

Scientists at Great Ormond Street Hospital and UCL Great Ormond Street Institute of Child Health (GOS ICH), have now used a technique, called base editing, to make precise genetic changes to T-cells obtained from a human donor, to restore their ability to seek out and kill cancer cells – see video.

Animals have been used to test this approach for safety and effectiveness, including as a therapy for leukaemia, and have also been important in the development of other gene editing technologies more widely, such as CRISPR-Cas9 (see EARA article).

Professor Waseem Qasim at GOS ICH said: “This is a great demonstration of how, with expert teams and infrastructure, we can link cutting edge technologies in the lab with real results in the hospital for patients.”


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