Phase 1 evaluation of CRISPR-CAR gene edited T cells in relapsed refractory B cell acute lymphoblastic leukaemia

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New treatments for leukaemia using gene therapy to re-program white bloods cells against cancer cells are showing a great deal of promise. In some trials, over 8 out of every 10 patient treated had a good response. Most of the trials so far have relied in collecting a patients own white cells and then using a disabled virus to fit new genes into a group of cells called T cells. This process then arms the cells against leukaemia, so when they are given back to the patients they can attack the disease. We recently showed that its possible not only to arm cells against leukaemia but also disarm them to stop side effects if they are taken from a healthy donor and transplanted to a patient without any matching. A technique called gene editing was used to to achieve this, and we are currently running a clinical trial to test the approach in more patients. In the meantime, improvements in the way we modify cells have led to the development of new versions of the treatment that we think are going to be more widely used. This project will test the new versions, made using CRISPR gene editing, to see if we can treat leukaemia in children where all other treatments have failed. We aim to treat 10 children over two years, and if they can be cleared of leukaemia, they will receive a vote marrow transplant to improve their chances of cure.