BRIEF RESEARCH REPORT article
Front. Immunol.
Sec. Cancer Immunity and Immunotherapy
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1602189
This article is part of the Research TopicPrecision Immunotherapy and Novel Target Discovery in Hematological MalignancyView all 12 articles
Drug repurposing of fostamatinib against cancer via potential cytotoxicity and immune checkpoint regulation
Provisionally accepted
- 1Wuhan First Hospital, Wuhan, Hebei Province, China
- 2Liuzhou Workers Hospital, Liuzhou, China
- 3First People's Hospital of Nanning, Nanning, Guangxi Zhuang Region, China
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Acute myeloid leukemia (AML), originating from myeloid hematopoietic stem/progenitor cells, is a malignant hematological disorder. Resistance to current treatments, especially in FLT3-ITD AML cases, urgently demands the development of novel therapeutics. In this study, we pinpointed fostamatinib, an orally delivered small molecule SYK inhibitor for chronic immune thrombocytopenia (ITP), as a promising candidate for drug repurposing. It effectively inhibited FLT3-ITD+ AML cell proliferation and induced leukemic cell apoptosis. Network pharmacology analysis further deciphered the associated pharmacological mechanism related to the PI3K-AKT signaling pathway. Moreover, fostamatinib downregulated the expression of immune checkpoints such as PD-L1 and CD47. Overall, this study provided a conceptual foundation for evaluating the advantages of drug repurposing in AML drug development.
Keywords: Fostamatinib, Cytotoxicity, Immune checkpoint, Network Pharmacology, drug repurposing
Received: 29 Mar 2025; Accepted: 12 May 2025.
Copyright: © 2025 Hu, Yin, Deng and Xu. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Kaifeng Deng, Liuzhou Workers Hospital, Liuzhou, China
Ning Xu, First People's Hospital of Nanning, Nanning, Guangxi Zhuang Region, China
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