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Cellular and Molecular Biology

Approval of DFMO for high-risk neuroblastoma patients demonstrates a step of success to target MYC pathway

British Journal of Cancer volume 130, pages 513–516 (2024)Cite this article

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Abstract

The “undruggable” MYC oncoproteins are deregulated in 70% human cancers. The approval of DFMO, an irreversible inhibitor of ornithine oxidase (ODC1) that is a direct transcriptional target of MYC, demonstrates that patients can benefit from targeting MYC activity via an indirect approach. However, the mechanism of action of DFMO needs further studies to understand how it works in post-immunotherapy neuroblastomas. Efforts to develop a more potent and safer drug to block MYC function will continue despite challenges.

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Fig. 1: Targeting MYC pathway as therapeutic approaches in cancer treatment.
Fig. 2: ODC1 knockout effect vs DFMO (eflornithine) effect.

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Acknowledgements

JY was supported by American Cancer Society-Research Scholar (130421-RSG-17-071-01-TBG, JY), National Cancer Institute (1R01CA229739-01, 1R01CA266600-01A1, JY), and the American Lebanese Syrian Associated Charities (ALSAC). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. JY is a Subject Editor for the British Journal of Cancer.

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  1. Department of Surgery, St Jude Children’s Research Hospital, 262 Danny Thomas Place, Memphis, TN, 38105, USA

    Jun Yang

  2. St Jude Graduate School of Biomedical Sciences, St Jude Children’s Research Hospital, Memphis, TN, 38105, USA

    Jun Yang

  3. Department of Pathology and Laboratory Medicine, College of Medicine, The University of Tennessee Health Science Center, 930 Madison Ave, Suite 500, Memphis, TN, 38163, USA

    Jun Yang

  4. College of Graduate Health Sciences, University of Tennessee Health Science Center, Memphis, TN, 38163, USA

    Jun Yang

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JY conducted the literature review, wrote and edited the paper.

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Correspondence to Jun Yang.

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Yang, J. Approval of DFMO for high-risk neuroblastoma patients demonstrates a step of success to target MYC pathway. Br J Cancer 130, 513–516 (2024). https://doi.org/10.1038/s41416-024-02599-6

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