New ‘off-the-shelf’ immunotherapy shows promise for treating high-risk childhood leukaemia

Acute lymphoblastic leukaemia is the most common cancer in children. While around 80% of children are cured with current treatments, cure rates for infants and children with high-risk forms of the disease remain around 50%. These patients are often treated with a therapy called CAR-T immunotherapy, which uses engineered versions of a patient’s own immune cells to fight cancer cells. This treatment is highly personalised, but can take weeks to prepare.

In this study, the research team, led by Professors Anindita Roy and Thomas Milne at the MRC Molecular Haematology Unit and Professor Anastasios Karadimitris at Imperial College London, tested a modified therapy called CAR-iNKT. This modified therapy uses a different type of cell to the standard CAR-T therapy called invariant natural killer cells (iNKT cells), which can be created from healthy donors and stored in advance, making them “off-the-shelf”. Having treatment readily available in this way can be especially important in high-risk leukaemia in children, where the cancer may be rapidly progressing.

For the new therapy, developed using funding from Cancer Research UK and Children with Cancer UK, researchers engineered CAR-iNKT cells to recognise two markers together on the high-risk leukaemia cells called CD19 and CD133. This two-target approach was highly effective when tested in mice; leukaemia cells were completely eradicated and mice remained free of leukaemia. In comparison, standard CAR-T therapy could only remove leukaemia for a few weeks before it returned.

Read the full story on the University of Oxford website.