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Lomitapide repurposing for treatment of malignancies: A promising direction
Hua-Tao Wu1,2, Bing-Xuan Wu1,2, Ze-Xuan Fang2,3
1Department of General Surgery, the First Affiliated Hospital of Shantou University Medical College, Shantou, 515041, China.
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.
Insights
Drug repurposing offers a cost-effective strategy for cancer therapy. Lomitapide, initially for hypercholesterolemia, shows potential anti-tumor effects by influencing cancer cell apoptosis, autophagy, and metabolism.
Area of Science:
- Oncology
- Pharmacology
- Biochemistry
Background:
- Drug development is lengthy and expensive.
- Drug repurposing accelerates the availability of treatments.
- Lomitapide is approved for familial hypercholesterolemia.
Purpose of the Study:
- To review lomitapide's potential as an anti-cancer agent.
- To explore lomitapide's anti-tumor mechanisms.
- To support repurposing lomitapide for cancer treatment.
Main Methods:
- Systematic literature review.
- Analysis of lomitapide's effects on cancer cell apoptosis.
- Investigation of lomitapide's impact on cancer cell autophagy.
- Examination of lomitapide's role in tumor cell metabolism.
Main Results:
- Lomitapide demonstrates potential anti-cancer properties.
- Mechanisms include modulation of apoptosis and autophagy.
- Lomitapide affects tumor cell metabolism.
Conclusions:
- Lomitapide exhibits promising anti-tumor functions.
- Repurposing lomitapide for cancer therapy is feasible.
- Further clinical investigation is warranted.

