Precision Medicine, Use of Cell Therapy

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

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 chemically converting letters of the DNA code which carry instructions for a specific protein.

The edited CAR T-cells can be given to a patient so that they quickly find and destroy T-cells in the body, including cancerous ones, after which the person can have a bone marrow transplant to restore their depleted immune system. Twenty-eight days after being given the treatment, Alyssa was in remission, researchers said, and was able to have a second bone marrow transplant.

Alyssa received the treatment at Great Ormond Street (Image: PA)

She is said to be “doing well at home” as she recovers and continues with follow-up monitoring at GOSH. It is hoped the research, due to be presented for the first time at the American Society of Haematology annual meeting in New Orleans in the US this weekend, could lead to new treatments and “ultimately better futures for sick children”.

Scientists aim to recruit up to 10 patients who have T-cell leukaemia and have exhausted all conventional options for the clinical trial into the new treatment. Medics at GOSH hope that if it is successful it can be offered to children earlier in their treatment when they are less sick and that it can be used for other types of leukaemia in future.

Girl, 13, ‘leukaemia-free’ after world-first use of cell engineering therapy - Wales Online: