Funded by the Little Princess Trust and administered by CCLG
Lead investigator: Prof Owen Williams, University College London Great Ormond Street Institute of Child Health
Award: £269,426.42
Awarded March 2026
The challenge
Acute myeloid leukaemia (AML) is a type of blood cancer which can affect children. In around 20% of cases, the cancer is caused by genetic mutations in a gene called KMT2A which result in the formation of KMT2A fusion genes. These abnormal genes help the cancer grow and make it harder to treat. Despite many improvements in treatments, only around half of these patients can be cured.
Research has shown that these fusion genes can use a molecule called menin to hijack blood cell machinery. This forces the blood cells to keep dividing and become locked into becoming AML cells. Drugs that block menin have now been developed and are showing exciting results, but the disease often comes back after the drug is stopped.
The project
Prof Owen Williams’ team at University College London hopes to create a combination treatment that will help the menin-blocking drug kill cancer cells – and ensure that the cancer does not come back. They have already identified a second altered process inside AML cells, which allows the cancer cells to use sugar as fuel to survive the drug. In early lab tests, blocking this process was able to help the menin-blocking drug kill cancer cells.
In this project, the researchers want to find the best way to do this in patients – finding a treatment that can safely and effectively block cancer cells from using sugar to survive. This would form part of a combination treatment with the menin-blocking drug that could be excellent at fighting AML.
The impact
By proving that this approach will kill the cancer cells more effectively while sparing normal blood cells, the researchers hope to improve the treatment of AML. Importantly, the original menin-blocking drug is already in clinical trials, so any promising results from this project could be more quickly introduced for children with AML. Prof Williams believes that this project will help bring a revolutionary new treatment for children and increase their chances of surviving their cancer.
The Little Princess Trust
This project was funded by The Little Princess Trust. They fund research projects in partnership with CCLG, combining CCLG's research funding and grant management expertise with The Little Princess Trust's fantastic fundraising to support world-class scientific research.