Review
Copyright ©The Author(s) 2019. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Stem Cells. Jul 26, 2019; 11(7): 398-420
Published online Jul 26, 2019. doi: 10.4252/wjsc.v11.i7.398
Targeting cancer stem cells in drug discovery: Current state and future perspectives
Fang-Yu Du, Qi-Fan Zhou, Wen-Jiao Sun, Guo-Liang Chen
Fang-Yu Du, Qi-Fan Zhou, Wen-Jiao Sun, Guo-Liang Chen, Key Laboratory of Structure-Based Drug Design and Discovery of Ministry of Education, Shenyang Pharmaceutical University, Shenyang 110016, Liaoning Province, China
Author contributions: All authors equally contributed to the conception and design of the study, literature review and analysis, drafting and critical revision of the manuscript and approved the final version of the manuscript.
Supported by Natural Science Foundation of Liaoning Province, No. 201602707.
Conflict-of-interest statement: The authors declare no potential conflicts of interest.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Corresponding author: Guo-Liang Chen, PhD, Key Laboratory of Structure-Based Drug Design and Discovery of Ministry of Education, Shenyang Pharmaceutical University, No. 103, Wenhua Road, Shenhe District, Shenyang 110016, Liaoning Province, China. chenguoliang@syphu.edu.cn
Telephone: +86-24-43520236
Received: February 20, 2019
Peer-review started: February 22, 2019
First decision: June 4, 2019
Revised: June 18, 2019
Accepted: June 27, 2019
Article in press: June 27, 2019
Published online: July 26, 2019
Processing time: 155 Days and 19.6 Hours
Abstract

In recent decades, cancer stem cells (CSCs) have been increasingly identified in many malignancies. CSC-related signaling pathways and their functions provide new strategies for treating cancer. The aberrant activation of related signaling pathways (e.g., Wnt, Notch, and Hedgehog pathways) has been linked to multiple types of malignant tumors, which makes these pathways attractive targets for cancer therapy. CSCs display many characteristic features, such as self-renewal, differentiation, high tumorigenicity, and drug resistance. Therefore, there is an urgent need to develop new therapeutic strategies to target these pathways to control stem cell replication, survival, and differentiation. Notable crosstalk occurs among different signaling pathways and potentially leads to compensatory escape. Therefore, multitarget inhibitors will be one of the main methods to overcome the drug resistance of CSCs. Many small molecule inhibitors of components of signaling pathways in CSCs have entered clinical trials, and some inhibitors, such as vismodegib, sonidegib, and glasdegib, have been approved. Tumor cells are susceptible to sonidegib and vismodegib resistance due to mutations in the Smo protein. The signal transducers and activators of transcription 3 (STAT3) inhibitor BBI608 is being evaluated in a phase III trial for a variety of cancers. Structural derivatives of BBI608 are the main focus of STAT3 inhibitor development, which is another strategy for CSC therapy. In addition to the potential pharmacological inhibitors targeting CSC-related signaling pathways, other methods of targeting CSCs are available, such as nano-drug delivery systems, mitochondrion targeting, autophagy, hyperthermia, immunotherapy, and CSC microenvironment targeting. In addition, we summarize the latest advances in the clinical development of agents targeting CSC-related signaling pathways and other methods of targeting CSCs.

Keywords: Cancer stem cells; Cancer stem cell-related signaling pathways; Nano-drug delivery system; Immunotherapy; Mitochondrion; Immunotherapy; Cancer stem cell microenvironment

Core tip: The review aims to introduce the field of cancer stem cells (CSCs) and the important signaling pathways in CSCs and present approved inhibitors as well as candidate drugs. Due to the complexity of the crosstalk among various signaling pathways, current strategies involve the development of multitarget inhibitors, combination therapy, and precision treatments based on the genetic characteristics of patients. Other methods of targeting CSCs are introduced as well, including nano-drug delivery systems, mitochondrion targeting, hyperthermia, immunotherapy, and CSC microenvironment targeting. However, this field remains in its infancy, and considerable research will be required to produce mature products that can contribute to curing cancer.