New Immunotherapy Shows Promise for Treating High-Risk Child Leukemia

The latest CDC data shows an increase in childhood cancer rates over the past decade, and with it, the need for quick and effective treatments continues to grow.

One promising advancement comes from a University of Oxford lab, where researchers have developed an “off-the-shelf” immunotherapy that could improve outcomes for babies and children with high-risk acute lymphoblastic leukemia—the most common childhood cancer today.

The new therapy is a modified version of pre-existing CAR-T immunotherapy, which uses engineered versions of a patient’s own immune cells to fight cancer. CAR-iNKT therapy instead harnesses a different type of immune cell called invariant natural killer (iNKT) cells. Because these can be created from healthy donors and stored in advance, the therapy can be used “off-the-shelf.” That’s a major improvement in a field where current CAR-T treatments take weeks to produce; delivering therapy faster may help contain the spread of cancer and improve survival.

Not only is CAR-iNKT therapy quicker to develop, but in a recent study published in the journal Blood it also proved to be more effective against high-risk cancers. The engineered CAR-iNKT cells were designed to recognize two markers on high-risk leukemia cells—CD19 and CD133. This two-target approach completely eradicated leukemia cells in mice, keeping them cancer-free. By contrast, standard CAR-T therapy cleared the cancer only temporarily, with the disease returning after a few weeks.

Researchers also found CAR-iNKT therapy may be particularly useful against leukemia cells that invade the space around the brain, as well as other difficult-to-treat areas such as the bone marrow and spleen. Blood cancer that persists in the brain is one of the major reasons children relapse from high-risk leukemia, so targeting these sites could allow young patients to remain cancer-free longer.

Natalina Elliott, PhD, a postdoctoral researcher and co-first author of the study, said the next step is to bring this potentially groundbreaking therapy to human patients. The team has received a Children and Young People’s Cancer Innovation Award from Cancer Research UK and Children with Cancer to further develop the treatment. With continued funding and research, they hope to move one step closer to a cure.