Our team has guided pre-clinical and clinical research resulting in regulatory approval of new drugs including oral azacitidine as maintenance therapy in AML (NEJM 2020) and venetoclax (JCO 2019 and Blood 2020). We designed pre-clinical studies that supported the first clinical trial combining venetoclax with intensive chemotherapy (Leukemia 2018 and J of Clin Oncol 2020). This study paved the way for venetoclax to be tested in younger patients by groups around the world.
Our lab conducted biomarkers studies guiding use of venetoclax in AML using patient samples from clinical trials (Blood 2020) in collaboration with MD Anderson, Roberts and Majewski from WEHI and identified the importance of TP53 mutation in venetoclax failure (Blood 2021) in collaboration with Huang, Kelly and Roberts lat WEHI and the role of BAX variants in venetoclax resistance (Blood 2022) in collaboration with the Huang, Roberts and Adams. We pioneered the combination of BH3-mimetics to target both BCL-2 and MCL1 (Leukemia 2019) in collaboration with Lessene and Servier. This work led to a first in human trial (NCT03672695).