A better way of delivering drugs for acute myeloid leukaemia

Project title: Improving outcomes for childhood AML using nanovector delivered therapy 

Funded by The Little Princess Trust and administered by CCLG
Lead investigator: Dr Allison Blair, University of Bristol
Award: £199,971.36 
Awarded March 2022 

Around 70 children in the UK are diagnosed with acute myeloid leukaemia (AML) each year, which is around 15% of all patients with childhood leukaemia. The normal treatments are don’t work as well for children with AML, with only 68% of children still alive after five years. The outcomes for children with relapsed AML are much worse and many do not survive.  

The normal treatments for AML can be very damaging to normal cells and major organs, like the heart. They also do not always reach the places in the body where leukaemia cells are. In addition, the drugs are given separately, normally on different days, which means that they are less likely to find and kill the same leukaemia cells. Increasing the amount of the drugs to kill the remaining leukaemia cells is not an option, as it would be even more damaging to the child’s body. In fact, in every five children who die with relapsed leukaemia, one will be because of the toxic treatments. We urgently need less dangerous treatment options for children with AML. 

The research team at the University of Bristol, led by Dr Allison Blair, have developed a new way to get the drugs to where they are most needed. They plan to package drugs together into tiny nanoparticles. These nanoparticles last longer in the body, so it should not only reduce damage to a child’s healthy cells, but also mean that all of the packaged drugs arrive at the leukaemia cells at the same time. This means that all of the drugs can work on the same leukaemia cells at the same time, which Dr Allison Blair hopes will improve survival and reduce side effects. Because the nanoparticles last longer in the body, they could even mean that lower doses of these toxic drugs can be used.