Precision medicines for paediatric acute myeloid leukaemia

Project title: Precision medicines for paediatric acute myeloid leukaemia 

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Professor Julie Irving, Newcastle University 
Award: £227,557.00 
Awarded March 2022 

Acute myeloid leukaemia (AML) is a type of blood cancer that causes the body’s white blood cells to grow out of control. It is the second most common children’s leukaemia but the cure rate is still low. We clearly need better drugs to cure more of these young patients. Many exciting new cancer drugs have proven effective in some adult cancers and could also work for paediatric AML but have not been properly tested in children.  

One exciting finding is that about a third of children have specific genetic alterations that cause the AML cells to grow and multiply too fast. There are new smart drugs called ‘MEK inhibitors’ that can turn stop this effect, which then makes the cancer cells die. We know from adult cancers that MEK inhibitors are more effective when used with other anti-cancer drugs. For children with AML, we do not know which type of MEK inhibitor will be the best one to use or which other drug will work best with it.  

The research team at Newcastle University, led by Professor Julie Irving, will use advanced technologies to find the MEK inhibitor drug combination that works best for children with AML. They will then create a data package that will help with getting the new drug combination into a clinical trial, where it can help real patients. The researchers will also use the same technologies to find other types of new drugs for another high-risk group of children where the driver of their AML is not known. Professor Julie Irving hopes to find effective new precision treatments for all children with high-risk AML that will improve their chances of leading long healthy lives.