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Review
. 2024 Jun 13;10(12):e32998.
doi: 10.1016/j.heliyon.2024.e32998. eCollection 2024 Jun 30.

Lomitapide repurposing for treatment of malignancies: A promising direction

Hua-Tao Wu  1   2 , Bing-Xuan Wu  1   2 , Ze-Xuan Fang  2   3 , Zheng Wu  2   3 , Yan-Yu Hou  2   3 , Yu Deng  1   2 , Yu-Kun Cui  2 , Jing Liu  2   3
Affiliations
Review

Lomitapide repurposing for treatment of malignancies: A promising direction

Hua-Tao Wu et al. Heliyon. .

Abstract

The development of novel drugs from basic science to clinical practice requires several years, much effort, and cost. Drug repurposing can promote the utilization of clinical drugs in cancer therapy. Recent studies have shown the potential effects of lomitapide on treating malignancies, which is currently used for the treatment of familial hypercholesterolemia. We systematically review possible functions and mechanisms of lomitapide as an anti-tumor compound, regarding the aspects of apoptosis, autophagy, and metabolism of tumor cells, to support repurposing lomitapide for the clinical treatment of tumors.

Keywords: Cancer; Lomitapide; Repurposing; Therapy; mTOR signaling.

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Conflict of interest statement

The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Figures

Fig. 1
Fig. 1
Structure of lomitapide. Compound ID: 9853053; Molecular Formula: C39H37F6N3O2; Molecular Weight: 693.7 [g/mol] [22].
Fig. 2
Fig. 2
The lipid-lowering molecular mechanism of lomitapide. Abbreviation: Apo B-apolipoprotein B, MTTP-microsomal triglyceride transfer protein, VLDL-very low-density lipoprotein, LDL-low-density lipoprotein.
Fig. 3
Fig. 3
The molecular mechanism of lomitapide suppressing malignancies.
Fig. 4
Fig. 4
The graphic abstract of lomitapide in malignancies.
See this image and copyright information in PMC

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