Projects we fund

Overview

Acute myeloid leukaemia (AML) is a rare type of childhood leukaemia. It is caused by the body overproducing myeloid cells, a type of white blood cell. CAR T-cell therapy is an exciting new type of cancer treatment. It works by instructing T cells from the body’s immune system to attack cancer cells that display a particular molecule on their surface. Research into CAR T-cell therapy so far has done an incredible job in improving survival rates for some cancer types, such as the more commonly occurring acute lymphoblastic leukaemia (ALL). However, right now there isn’t an equivalent CAR T-cell therapy for AML, which is more difficult to treat this way. One of the reasons for this difficulty is that AML cells do not all look alike. This means that a CAR T-cell therapy that targets only one molecule might not kill all the AML cells. In addition, the stem cells in bone marrow, which produce our blood, can look similar to AML cells. This means that a CAR T-cell therapy for AML might attack these healthy stem cells, which could cause devastating side effects. Dr Macarena Oporto Espuelas aims to develop a new type of CAR T-cell therapy for relapsed or treatment-resistant AML that recognises and kills more of the cancerous cells while leaving more the healthy stem cells unharmed. She will analyse the gene activity of different AML cells to identify up to three targets that are present in AML cells, but not healthy stem cells. This will allow her to engineer T cells that seek out these targets in combination. Using a combination of targets helps the T cells to recognise and kill more AML cells without mistaking healthy stem cells as cancerous.

What difference will this project make?

CAR T-cell therapy is a type of immunotherapy that uses the body’s own immune system to find and kill cancer cells. Unlike chemotherapy, which typically attacks all fast-growing cells, CAR T-celltherapy tries to only target cancer cells, leaving most healthy cells unaffected. CAR T-cell therapy has been particularly successful in treating children with ALL, however, for some other cancers progress has been slower. Right now, there isn’t an equivalent CAR T-cell therapy for childhood AML. The five-year survival rate for this cancer is around 70% and has improved little in the past twenty years. Furthermore, the treatments that are available can have gruelling and long-lasting side-effects. If successful, Dr Macarena Oporto Espuelas’ research could develop a new CAR T-cell therapy for relapsed and hard-to-treat AML that is more effective and less toxic than existing treatments. This would increase survival, quicken recovery and improve long term health for young patients.