Targeting FSP1 triggers ferroptosis in lung cancer
Emerging evidence indicates that cancer cells are susceptible to ferroptosis, a form of cell death that is triggered by uncontrolled lipid peroxidation<sup>1-3</sup>. Despite broad enthusiasm about harnessing ferroptosis as a novel anti-cancer strategy, whether ferroptosis is a barrier to tumorigenesis and can be leveraged therapeutically remains unknown<sup>4,5</sup>. Here, using genetically engineered mouse models of lung adenocarcinoma, we performed tumour-specific loss-of-function studies of two key ferroptosis suppressors, GPX4<sup>6,7</sup> and ferroptosis suppressor protein 1 (FSP1)<sup>8,9</sup>, and observed increased lipid peroxidation and robust suppression of tumorigenesis, suggesting that lung tumours are highly sensitive to ferroptosis. Furthermore, across multiple pre-clinical models, we found that FSP1 was required for ferroptosis protection in vivo, but not in vitro, underscoring a heightened need to buffer lipid peroxidation under physiological conditions. Lipidomic analyses revealed that Fsp1-knockout tumours had an accumulation of lipid peroxides, and inhibition of ferroptosis with genetic, dietary or pharmacological approaches effectively restored the growth of Fsp1-knockout tumours in vivo. Unlike GPX4, expression of FSP1 (also known as AIFM2) was prognostic for disease progression and poorer survival in patients with lung adenocarcinoma, highlighting its potential as a viable therapeutic target. To this end, we demonstrated that pharmacologic inhibition of FSP1 had significant therapeutic benefit in pre-clinical lung cancer models. Our studies highlight the importance of ferroptosis suppression in vivo and pave the way for FSP1 inhibition as a therapeutic strategy for patients with lung cancer.
Authors
- Mari N. Nakamura
- Katherine Wu (ORCID: https://orcid.org/0000-0002-0472-0166)
- Diane M. Simeone (ORCID: https://orcid.org/0000-0001-5142-3087)
- Marcus Conrad (ORCID: https://orcid.org/0000-0003-1140-5612)
- Sungjin Chung (ORCID: https://orcid.org/0000-0002-9886-8339)
- Jozef P. Bossowski
- Tae Ha Kim (ORCID: https://orcid.org/0000-0001-5182-8578)
- Mariana Camargo Silva Mancini (ORCID: https://orcid.org/0000-0002-2755-0032)
- Toshitaka Nakamura (ORCID: https://orcid.org/0000-0002-4248-2058)
- Ray Pillai (ORCID: https://orcid.org/0000-0002-8335-3463)
- Sahith Rajalingam (ORCID: https://orcid.org/0000-0003-3669-3729)
- Yun Pyo Kang (ORCID: https://orcid.org/0000-0002-1149-0880)
- Thales Papagiannakopoulos (ORCID: https://orcid.org/0000-0002-2251-1624)
- David B. Shackelford (ORCID: https://orcid.org/0000-0002-8270-898X)
- Yuan Hao (ORCID: https://orcid.org/0000-0002-4031-7868)
- Lidong Wang (ORCID: https://orcid.org/0000-0003-4204-3255)
- Seon Min Kim
- Mingqi Han (ORCID: https://orcid.org/0000-0003-2070-5967)
- Alec Vaughan (ORCID: https://orcid.org/0000-0002-0154-8911)
- Aikaterini Ziogou (ORCID: https://orcid.org/0009-0000-0028-6748)
Institutions
- Seoul National University (KR)
- University of California, Los Angeles (US)
- VA NY Harbor Healthcare System (US)
- University of California San Diego (US)
- NYU Langone Health (US)
- Target (Germany) (DE)
- UCLA Jonsson Comprehensive Cancer Center
- New York University (US)
- Technical University of Munich (DE)
Publication Details
- Journal
- Nature
- Published
- 2025-11-05
- DOI
- https://doi.org/10.1038/s41586-025-09710-8
- Citations
- 27
- Primary Topic
- Ferroptosis and cancer prognosis
- Type
- article
- Field-Weighted Citation Impact
- 41.00