A trial looking at gene therapy for acute myeloid leukaemia and chronic myeloid leukaemia

Doctors often treat acute myeloid leukaemia (AML) and chronic myeloid leukaemia (CML) with chemotherapy, and sometimes a stem cell transplant . But sometimes the leukaemia comes back.

The research team wanted to find out if T cell receptor (TCR) gene therapy is useful for people in this situation.  

Doctors remove specific immune system cells (called T cells) from a person’s blood sample. Then they change (modify) the cells in the laboratory. They add a gene to the cells to help them recognise a protein called WT1. The doctors then give the person back their T cells through a drip into a vein.

Abnormal blood cells such as leukaemia cells often have a high level of WT1. Other cells in the body usually have a very low level of WT1. Researchers hoped the modified T cells would recognise the WT1 protein and attack the leukaemia cells.

Everybody taking part had WT1 TCR gene therapy. Some people had a lower dose. And some people had a higher dose.

The main aims of the trial were to find out:

• the best dose of modified T cells to give
• more about the side effects
• whether the modified T cells recognise leukaemia cells