Making an ‘off-the-shelf’ CAR-T cell treatment for children with solid cancers

CAR-T treatment has been shown to be very effective in childhood leukaemia. It uses genetically engineered immune cells to fight cancer - doctors extract the cells from a cancer patient’s own immune system and modify them so they can recognise and kill cancer cells. Currently, it has to use a patient’s own immune cells so that the body doesn’t fight the treatment, a bit like when the body rejects organ transplants.

Early results in clinical trials, showed that CAR-T cells could lead to complete remission in children with chemotherapy-resistant leukaemia that had been considered incurable. Several CAR-T treatments have now been approved for childhood and adult blood cancers. CAR-T treatments for children’s solid cancers have also shown promise in recent clinical trials for neuroblastoma and brain tumours.

However, there are significant disadvantages to the current way CAR-T cells are produced. The treatments take weeks to make and can only be made in specialist centres or by pharmaceutical companies. This means that any manufacturing failures may mean patients need to repeat procedures, and that it is extremely expensive to the NHS. Crucially, patients’ cancers can progress whilst they wait for their treatment to be created and authorised.

In this research project, Professor John Anderson aims to address these problems. His team at UCL Great Ormond Street Institute of Child Health hope to create an ‘off-the-shelf’ CAR-T cell treatment that can be made in bulk. This means the treatment could be stored, ready for use whenever it is needed. However, it would mean that the CAR-T cells would need to be created from general blood donations, rather than from a patient’s own immune cells.

To develop their treatment, Professor Anderson’s team will use a different type of T-cell to standard CAR-T cells. They are also using a new technique to genetically engineer them. This will allow them to prevent the CAR-T cells from being rejected by the patient’s immune system, and to reduce the toxicity of the treatment. If their method is successful, it will be more cost-effective, allowing CAR-T treatment to become more widely available to children with cancer.