World-first use of base-edited cells to treat ‘incurable’ leukaemia
A patient with ‘incurable’ T-cell leukaemia has been treated with base-edited T-cells in the world’s first reported use of a base-edited therapy.
The patient, 13-year-old Alyssa from Leicester, was diagnosed with T-cell acute lymphoblastic leukaemia in 2021.
She was treated with all current conventional therapies for her cancer, including chemotherapy and a bone marrow transplant, but unfortunately her disease came back and there were no further treatment options.
In May, Alyssa was admitted to the Bone Marrow Transplant (BMT) Unit at Great Ormond Street Hospital (GOSH) to receive genetically modified chimeric antigen receptor (CAR) T-cells.
The CAR T-cells originally came from a healthy donor.
These cells had been edited using new base-editing technology to allow them to hunt down and kill the cancerous T-cells without attacking each other.
Researchers are presenting the data for the first time at the American Society of Haematology annual meeting in New Orleans, US, this weekend.
Trials have continued using clustered regularly interspaced short palindromic repeats (CRISPR) or CRISPR associated protein 9 (cas9) techniques.
However, it’s been more difficult to treat T-cell leukaemia in this way. This is because T-cells designed to recognise and attack cancerous T-cells also end up killing each other during the manufacturing process in the lab.
Multiple additional changes have now been added by the GOSH team to generate banks of ‘universal’ anti-T-cell CAR T-cells.
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