What is AML?

Acute Myeloid Leukaemia is the second most common leukaemia (cancer of the blood) of childhood. Current treatment is very intensive and based on chemotherapy given over a period of about 6 months or longer. Treatment often requires inpatient admission for the majority of this time and in high risk disease can include a bone marrow transplant. During therapy, side-effects of treatment will result in hair loss, organ toxicity, and a significant risk of life-threatening infections. The psychological and social impact of undergoing treatment is a significant hardship for both the child and their families. Patients who are cured may go on to suffer long-term side effects including cardiac failure and a risk of a second cancer developing, due to chemotherapy that they received. Unlike ALL (the most common childhood leukaemia where the majority of patients are cured) in AML many patients do not respond fully to treatment and die of their disease.

Acute Myeloid Leukaemia is currently treated with chemotherapy based around 3 main drugs(Cytarabine, Doxorubicin, and Etoposide). Despite multinational research over the last 20years, no new drugs have been developed that have become clinically widely used for the treatment of AML. Therefore a major challenge is to find new drugs which are active in killing AML cells or which make AML cells more sensitive to the existing drugs used.