Funded by the Little Princess Trust and administered by CCLG
Lead investigator: Dr Cristina Pina, Brunel University London
Award: £45,412.34
Awarded July 2023
Acute myeloid leukaemia (AML) is a type of blood cancer that affects white blood cells. Although fortunately rare, leukaemia is the most common cancer in babies and toddlers, and half of children in this age group diagnosed with leukaemia have AML. Some forms of AML, which are only found in babies, are very difficult to study and therefore we don’t know how best to treat them. Because these forms of AML are rare, it is challenging to collect enough patient cancer cells to work on, and the cells have characteristics which are difficult to recreate in the lab.
Dr Cristina Pina’s lab at Brunel University London studies the most common form of AML that is only found in babies, called MNX1 leukaemia. This form has a genetic error where genetic material has been swapped between chromosomes leading to a gene, called MNX1, not working properly. Her team has successfully created two different models for MNX1 leukaemia: a cell line which can re-create the swapping of genetic material between chromosomes, and another model which forms a 3D model that mimics how the cancer would behave in an embryo.
In this project, the Dr Pina’s team have two goals, both of which depend on their new models of MNX1 leukaemia:
- Look for new treatments: The researchers will use the cell line to screen drugs and look for those that can stop the leukaemia cells from growing, kill them, or modify them to make them lose their cancerous properties. They are deliberately using drugs that have already been approved by the regulators in a variety of diseases, including cancer, to minimise unexpected and unwanted toxic effects.
- Validate potential treatments: Any promising drugs from the first part of the project will be tested in the 3D embryonic model system to see how they may affect development. Wherever the information is available, the researchers will prioritise drugs known to be safe for children. We will also look at these drugs in combination and check if the combinations are better at eliminating the leukaemia cells.
By the end of the project, Dr Pina hopes to have selected 3-5 drugs to test further with the hopes of producing enough data to consider clinical trials. The ultimate goal of this work is to find a targeted therapy for MNX1 leukaemia where chemotherapy alone has proven ineffective.