Revolutionary genetic treatment cures 13-year-old's 'incurable' cancer

11.12.2022 - 13:49 / dailyrecord.co.uk

A 13-year-old girl who relapsed with a particular type of blood cancer is now leukaemia-free after a world-first use of what scientists have described as the most sophisticated cell engineering to date.

Alyssa, whose family did not wish to give their surname, said she felt that taking the experimental new treatment for the disease would help others, adding “of course I’m going to do it”. Scientists said that without the treatment, which came after chemotherapy and an initial bone marrow transplant failed to clear her cancer, her only next step would have been palliative care.

Speaking about the revolutionary new therapy, Alyssa said: “Once I do it, people will know what they need to do, one way or another, so doing this will help people – of course I’m going to do it.” The teenager, from Leicester, received base-edited T-cells in the first ever use of a base-edited cell therapy at Great Ormond Street Hospital for Children (GOSH).

These pre-manufactured cells, from a healthy volunteer donor, were edited using new technology to allow them to hunt down and kill cancerous T-cells without attacking each other. T-cells are a type of white blood cell which move around the body to find and destroy defective cells.

Alyssa, who was diagnosed with T-cell acute lymphoblastic leukaemia (T-ALL) in 2021, was given all the conventional treatments including chemotherapy and a bone marrow transplant, but the disease returned. She then became the first patient enrolled onto a new clinical trial, funded by the Medical Research Council, during which she was given universal CAR (Chimeric Antigen Receptor) T-cells that had been pre-manufactured from a healthy volunteer donor in May this year.

The researchers described base-editing as