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A venetoclax bench-to-bedside story

Inhibition of the anti-apoptotic protein BCL-2 has emerged as a highly effective treatment for acute myeloid leukemia; approved lower-intensity venetoclax combination therapies are now being rapidly incorporated into an improved standard of care for this cancer. Here we recount an abbreviated history of venetoclax for acute myeloid leukemia, focusing on a selection of key studies along the path from development into the clinic.

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Fig. 1: An overview of BCL-2 family proteins and AML.

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Acknowledgements

C.D.D. and M.Y.K. are funded by US National Institutes of Health/National Cancer Institute grant R01CA235622 (principal investigator, M.Y.K.; 17 June 2019 to 31 May 2024). This research is supported in part by the MD Anderson Cancer Center Support Grant P30 CA016672 and the MD Anderson Cancer Center Leukemia SPORE CA100632.

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Correspondence to Courtney D. DiNardo.

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C.D.D. reports research funding from Abbvie, Agios, Calithera, Cleave, BMS/Celgene, Daiichi-Sankyo, ImmuneOnc and Loxo; advisory/consulting for Abbvie, Agios, Celgene/BMS, Daiichi Sankyo, ImmuneOnc, Novartis and Takeda; and stock options from Notable Labs. M.Y.K. reports research funding from AbbVie, Genentech, Eli Lilly, Cellectis, Calithera, Ablynx, Stemline Therapeutics, Sanofi, Rafael and Forty-Seven; advisory/consulting for AbbVie, Genentech, F. Hoffmann–La Roche, Cellectis, Stemline Therapeutics, Kisoji and Forty-Seven; and stock options/royalties from Reata Pharmaceuticals.

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DiNardo, C.D., Konopleva, M.Y. A venetoclax bench-to-bedside story. Nat Cancer 2, 3–5 (2021). https://doi.org/10.1038/s43018-020-00165-6

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