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Updated: May 19, 2026
In Vivo Inhibition of MicroRNA to Decrease Tumor Growth in Mice
Published on: August 23, 2019
Triparanol suppresses human tumor growth in vitro and in vivo
Xinyu Bi1, Xingpeng Han, Fang Zhang
1Department of Abdominal Surgical Oncology, Lab of Abdominal Surgical Oncology, Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100021, China.
Abstract:
Despite the improved contemporary multidisciplinary regimens treating cancer, majority of cancer patients still suffer from adverse effects and relapse, therefore posing a significant challenge to uncover more efficacious molecular therapeutics targeting signaling pathways central to tumorigenesis. Here, our study have demonstrated that Triparanol, a cholesterol synthesis inhibitor, can block proliferation and induce apoptosis in multiple human cancer cells including lung, breast, liver, pancreatic, prostate cancer and melanoma cells, and growth inhibition can be rescued by exogenous addition of cholesterol. Remarkably, we have proved Triparanol can significantly repress Hedgehog pathway signaling in these human cancer cells. Furthermore, study in a mouse xenograft model of human lung cancer has validated that Triparanol can impede tumor growth in vivo. We have therefore uncovered Triparanol as potential new cancer therapeutic in treating multiple types of human cancers with deregulated Hedgehog signaling.
Insights
Triparanol, a cholesterol synthesis inhibitor, effectively inhibits cancer cell proliferation and induces apoptosis. This compound also represses Hedgehog signaling, showing promise as a novel cancer therapeutic.
Area of Science:
- Oncology
- Molecular Biology
- Biochemistry
Background:
- Cancer treatment faces challenges with adverse effects and relapse.
- Novel molecular therapeutics targeting tumorigenesis pathways are needed.
- Cholesterol synthesis and Hedgehog signaling are implicated in cancer progression.
Purpose of the Study:
- To investigate Triparanol's efficacy against various human cancer cells.
- To determine Triparanol's effect on Hedgehog pathway signaling.
- To evaluate Triparanol's anti-tumor activity in vivo.
Main Methods:
- Treatment of multiple human cancer cell lines (lung, breast, liver, pancreatic, prostate, melanoma) with Triparanol.
- Assessment of cell proliferation and apoptosis.
- Rescue experiments with exogenous cholesterol.
- Analysis of Hedgehog pathway signaling.
- In vivo studies using a mouse xenograft model of human lung cancer.
Main Results:
- Triparanol blocked proliferation and induced apoptosis in diverse cancer cells.
- Cholesterol addition rescued Triparanol-induced growth inhibition.
- Triparanol significantly repressed Hedgehog pathway signaling.
- In vivo studies demonstrated Triparanol impeded lung cancer tumor growth.
Conclusions:
- Triparanol demonstrates broad-spectrum anti-cancer activity.
- Triparanol's mechanism involves inhibition of cholesterol synthesis and Hedgehog signaling.
- Triparanol represents a potential therapeutic agent for cancers with deregulated Hedgehog signaling.
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