The Little Princess Trust is funding research at Queen’s University Belfast into new approaches to target paediatric Acute Myeloid Leukaemia (AML).

Leukaemia is the most common childhood cancer and paediatric AML can affect any age group from new-born infants to young people aged 18–25 years. Around 70 new cases are diagnosed every year in the UK, which accounts for 15 per cent of children’s leukaemia cancer cases.

The patients with paediatric AML most at risk are children aged below two years; the risk in children aged two to nine years is lower; and it then rises through the adolescent years, with cases higher in boys than in girls.

The five-year survival rate for paediatric AML is very poor at approximately only 60 per cent of those diagnosed. This is compounded by using treatments with severe side effects for young patients including vomiting and hair loss.

Researchers at the Patrick G Johnston Centre for Cancer Research at Queen’s have identified a new drug combination which could be more time and cost effective in fighting paediatric AML and could also spare young patients the harsh side effects from treatment.

Professor Ken Mills, project lead at Queen’s University Belfast said, “Traditionally a generic combination of chemotherapy drugs and steroid medicines is administered to treat patients with paediatric AML. Our research aimed to provide a more targeted therapy. We found that the programmed cell death pathway was a potential target and identified that the specific drug combination of ABT-737, a Bcl-2 family inhibitor with Purvalanol A, a CDK inhibitor, together were a potential targeted therapy for AML patients with molecular abnormalities of the MLL and FLT3 genes.

“Our findings have the potential to reduce the cost, resource-intensity and time associated with the identification of novel combination therapies. This could reduce the time of drug development and enable previously untested and novel drug combinations to be tested and identified, contributing to a more rapid implementation into the clinic. This would overall improve outcomes for patients with paediatric AML.”

The Little Princess Trust’s funding is administered by the Children’s Cancer and Leukaemia Group.

Phil Brace, chief executive officer of The Little Princess Trust commented, “We are very pleased to have been able to fund this vital work. Although paediatric AML is relatively rare, there are still a significant number of children who are affected by the disease. Therefore, the news of any advances which could potentially lead to kinder and more effective treatments to improve their chance of survival gives significant hope for the future.”

The findings will next be validated through a clinical trial process. The full paper is available in the journal, Scientific Reports: https://www.nature.com/articles/s41598-020-75453-3.