Lomitapide as a Novel Therapeutic Agent in Breast Cancer: Mechanism and Potential Implications.

INTRODUCTION

Breast cancer treatment remains challenged by molecular heterogeneity and drug resistance, highlighting the need for new therapeutic strategies. This study aimed to discover potential anti-breast cancer agents using a drug repurposing strategy.

METHODS

We screened a library of 192 FDA-approved drugs and identified the lipid-lowering agent lomitapide as a candidate anti-breast cancer compound. Its biological effects were evaluated in MDA-MB-231 and MCF-7 cells, and potential molecular targets were investigated through virtual screening and experimental validation.

RESULTS

Lomitapide significantly inhibited cell proliferation, invasion, and migration. Mechanistically, lomitapide directly interacted with ER-α and PPARγ. Moreover, it enhanced the sensitivity of MCF-7 cells to tamoxifen.

DISCUSSION: Lomitapide suppresses breast cancer progression through multiple mechanisms and may improve endocrine sensitivity.

CONCLUSION

Lomitapide is a novel repurposed agent for breast cancer treatment. ER-α and PPARγ are potential therapeutic targets.