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.
This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
BH3 mimetics augment cytotoxic T cell killing of acute myeloid leukemia via mitochondrial apoptotic mechanism
Cell Death Discovery Open Access 26 March 2025
-
Feasibility and Safety of Targeting Mitochondria Function and Metabolism in Acute Myeloid Leukemia
Current Pharmacology Reports Open Access 04 October 2024
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$32.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 digital issues and online access to articles
$119.00 per year
only $9.92 per issue
Buy this article
- Purchase on SpringerLink
- Instant access to the full article PDF.
USD 39.95
Prices may be subject to local taxes which are calculated during checkout

References
Certo, M. et al. Cancer Cell 9, 351–365 (2006).
Ashkenazi, A., Fairbrother, W. J., Leverson, J. D. & Souers, A. J. Nat. Rev. Drug Discov. 16, 273–284 (2017).
Oltersdorf, T. et al. Nature 435, 677–681 (2005).
Roberts, A. W. et al. J. Clin. Oncol. 30, 488–496 (2012).
Souers, A. J. et al. Nat. Med. 19, 202–208 (2013).
Pan, R. et al. Cancer Discov. 4, 362–375 (2014).
Niu, X. et al. Clin. Cancer Res. 22, 4440–4451 (2016).
Bogenberger, J. M. et al. Leukemia 28, 1657–1665 (2014).
Konopleva, M. et al. Cancer Discov. 6, 1106–1117 (2016).
DiNardo, C. D. et al. Blood 133, 7–17 (2019).
Wei, A. H. et al. J. Clin. Oncol. 37, 1277–1284 (2019).
Wei, A. H. et al. Blood 135, 2137–2145 (2020).
DiNardo, C. D. et al. N. Engl. J. Med. 383, 617–629 (2020).
DiNardo, C. D. et al. Blood 135, 791–803 (2020).
Zhang, H. et al. Nat. Cancer 1, 826–839 (2020).
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.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
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.
Rights and permissions
About this article
Cite this article
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
Published:
Version of record:
Issue date:
DOI: https://doi.org/10.1038/s43018-020-00165-6
This article is cited by
-
BH3 mimetics augment cytotoxic T cell killing of acute myeloid leukemia via mitochondrial apoptotic mechanism
Cell Death Discovery (2025)
-
Conventional chemotherapy: millions of cures, unresolved therapeutic index
Nature Reviews Cancer (2024)
-
Immunosuppressive microenvironment in acute myeloid leukemia: overview, therapeutic targets and corresponding strategies
Annals of Hematology (2024)
-
Feasibility and Safety of Targeting Mitochondria Function and Metabolism in Acute Myeloid Leukemia
Current Pharmacology Reports (2024)
-
One year of Nature Cancer
Nature Cancer (2021)