3133 – DETERMINING EFFECTIVE DRUG COMBINATIONS IN PAEDIATRIC AML

• Published in: Experimental hematology 2022, Vol.111, p.S111-S111
• Main Authors: McGill, Áine, Cairns, Lauren, Prise, Kevin, Mills, Ken
• Format: Article
• Language: English
• ISSN: 0301-472X; 1873-2399
• DOI: 10.1016/j.exphem.2022.07.189

Paediatric acute myeloid leukaemia (pAML) accounts for ∼25% of childhood leukaemia diagnoses. Despite vast improvements in childhood cancer therapies, overall survival for pAML has not exceeded 70%. Alongside this, many of these pAML survivors experience treatment-induced side-effects which significantly affect their quality of life. Since 1980, only 11 drugs have been FDA approved for the treatment of paediatric cancers, therefore improving existing treatments is at the forefront of current research. Drug repurposing is a promising approach to address this problem as there is a rapid bench to bedside turn around and most FDA approved compounds have well-reported toxicity data. Combination therapies have the potential to be a superior approach to conventional monotherapies as the hallmarks of cancer are largely inter-linked. This suggests that combination therapies with variable target pathways are an apt approach to both limiting the incidence of relapse and to decrease the need for high-dose treatment. This study aims to identify novel drug combinations for paediatric AML that are just as effective but less toxic than the current standard of care treatments.