12 Dec 2022 Research

Widespread coverage of new leukaemia therapy shows promise in first trial patient

As you may have seen in the news over the weekend, a clinical trial investigating a new leukaemia treatment has shown promising results. As part of the trial, funded by the Medical Research Council, 13 year old Alyssa, who had exhausted all other treatment options for T-cell acute lymphoblastic leukaemia (T-ALL), was given a new type of CAR-T therapy in a world-first.

CAR-T therapy uses the immune system to kill cancer cells. It works by taking T cells from the blood and genetically modifying them, so they are better at recognising, finding and killing cancer cells, and then putting them back into the blood to eradicate the cancer.

Conventional CAR-T therapy can’t be used for treating T cell leukaemia, because the T cells are designed to attack other T cells, so the cells would end up attacking each other.

In this trial, a new technique called ‘base editing’ has been used, where the T cells have been modified, so that they are essentially shielded from other T cells and can go on to target and kill cancer cells.

Alyssa is the first patient in the world to receive this type of base-edited cell therapy. 28 days after treatment her cancer was in remission and she went on to have a second bone marrow transplant. Six months later, Alyssa’s leukaemia is still in remission, but she will need to be monitored closely over time.

The clinical trial is continuing to recruit up to 10 patients with T-cell leukaemia who have exhausted other treatment options. We wish Alyssa and her family all the very best and look forward to following the progress of this ground-breaking trial over the coming years.

Find out about T-ALL research we’re funding.