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A drug from poison: how the therapeutic effect of arsenic trioxide on acute promyelocytic leukemia was discovered
Open accessAbstract
It is surprising that, while arsenic trioxide (ATO) is now considered as "the single most active agent in patients with acute promyelocytic leukemia (APL)", the most important discoverer remains obscure and his original papers have not been cited by a single English paper. The discovery was made during the Cultural Revolution when most Chinese scientists and doctors struggled to survive. Beginning with recipes from a countryside practitioner that were vague in applicable diseases, Zhang TingDong and colleagues proposed in the 1970s that a single chemical in the recipe is most effective and that its target is APL. More than 20 years of work by Zhang and colleagues eliminated the confusions about whether and how ATO can be used effectively. Other researchers, first in China and then in the West, followed his lead. Retrospective analysis of data from his own group proved that APL was indeed the most sensitive target. Removal of a trace amount of mercury chloride from the recipe by another group in his hospital proved that only ATO was required. Publication of Western replication in 1998 made the therapy widely accepted, though neither Western, nor Chinese authors of English papers on ATO cited Zhang's papers in the 1970s. This article focuses on the early papers of Zhang, but also suggests it worth further work to validate Chinese reports of ATO treatment of other cancers, and infers that some findings published in Chinese journals are of considerable value to patients and that doctors from other countries can benefit from the clinical experience of Chinese doctors with the largest population of patients.
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“In addition, arsenic trioxide (a poison) and all-trans retinoic acid (a metabolite of vitamin A) are examples of other chemical compounds19 that were approved by the FDA in 2000 for the treatment of acute promyelocytic leukemia.”
From Drug repurposing for cancer therapy (2024) · cites this paper as [19]
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Metadata
- Authors
- Rao, Y., Li, R., & Zhang, D.
- Affiliations
- Peking University (CN)
- Corresponding author
- Rao, Yi
- ORCID
- Rao, Yi
- Venue
- Science China Life Sciences
- Published
- 2013-05-06
- Volume
- 56
- Issue
- 6
- Pages
- 495-502
- ISSN
- 1674-7305 (ISSN-L)
- 1869-1889
- OpenAlex
- W2092247417
- Cited by
- 58
- References
- 40
- Citations by year
- 2026: 1 · 2025: 3 · 2024: 5 · 2023: 5 · 2022: 2 · 2021: 8 · 2020: 9 · 2019: 3 · 2018: 5 · 2017: 5 · 2016: 3 · 2015: 4 · 2014: 4 · 2013: 1
- FWCI
- 0.38
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Science China Life Sciences (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinoids in leukemia and cellular processes
- Health Sciences › Medicine › Pharmacology (medical) › Historical Medical Research and Treatments
- Social Sciences › Arts and Humanities › History and Philosophy of Science › History of Science and Medicine
- Keywords
- arsenic trioxide, acute promyelocytic leukemia, history of drug discovery, traditional Chinese medicine, mercuric chloride, anticancer drug development, citation analysis, Chinese medical literature
- MeSH terms
- Arsenic Trioxide, Antineoplastic Agents, Arsenicals, China, Humans, Medicine, Chinese Traditional, Oxides, Remission Induction, Leukemia, Promyelocytic, Acute, Treatment Outcome, Asian People, Drug Discovery
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