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Updated: May 6, 2026

Quantitative PCR-based Assay to Measure Sonic Hedgehog Signaling in Cellular Model of Ciliogenesis
Published on: January 31, 2025
Targeting hedgehog signaling in cancer: research and clinical developments
Jingwu Xie1, Christopher M Bartels, Scott W Barton
1Wells Center for Pediatric Research, Division of Hematology and Oncology, Department of Pediatrics, Indiana University Simon Cancer Center, Indiana University, Indianapolis, IN, USA.
Abstract:
Since its first description in Drosophila by Drs Nusslein-Volhard and Wieschaus in 1980, hedgehog (Hh) signaling has been implicated in regulation of cell differentiation, proliferation, tissue polarity, stem cell maintenance, and carcinogenesis. The first link of Hh signaling to cancer was established through studies of Gorlin syndrome in 1996 by two independent teams. Later, it was shown that Hh signaling may be involved in many types of cancer, including skin, leukemia, lung, brain, and gastrointestinal cancers. In early 2012, the US Food and Drug Administration approved the clinical use of Hh inhibitor Erivedge/vismodegib for treatment of locally advanced and metastatic basal cell carcinomas. With further investigation, it is possible to see more clinical applications of Hh signaling inhibitors. In this review, we will summarize major advances in the last 3 years in our understanding of Hh signaling activation in human cancer, and recent developments in preclinical and clinical studies using Hh signaling inhibitors.
Insights
Hedgehog (Hh) signaling is crucial in cell development and linked to various cancers. Recent research highlights advances in understanding Hh signaling in cancer and developing Hh inhibitors for treatment.
Area of Science:
- Molecular Biology
- Oncology
- Developmental Biology
Background:
- Hedgehog (Hh) signaling, first described in Drosophila, regulates key cellular processes including differentiation, proliferation, and stem cell maintenance.
- Hh signaling has been linked to various human cancers, including skin, lung, brain, and gastrointestinal malignancies.
- The first Hh signaling inhibitor, vismodegib, was approved in 2012 for basal cell carcinoma, indicating therapeutic potential.
Purpose of the Study:
- To review recent advances (last 3 years) in understanding Hh signaling activation in human cancer.
- To summarize developments in preclinical and clinical studies of Hh signaling inhibitors for cancer treatment.
Main Methods:
- Literature review of recent scientific publications.
- Analysis of preclinical data on Hh signaling inhibitors.
- Evaluation of clinical trial outcomes for Hh signaling inhibitors.
Main Results:
- Significant progress has been made in elucidating the role of Hh signaling in diverse cancer types.
- Several Hh signaling inhibitors have shown promise in preclinical models and early-phase clinical trials.
- Understanding of Hh pathway activation mechanisms in cancer has expanded.
Conclusions:
- Hh signaling is a critical pathway in human carcinogenesis and a viable target for cancer therapy.
- Continued research into Hh signaling inhibitors may lead to new therapeutic strategies for various cancers.
- Further clinical investigation is warranted to optimize the use of Hh inhibitors in oncology.
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