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Updated: May 8, 2026

Cell Population Analyses During Skin Carcinogenesis
Published on: August 21, 2013
Profile of vismodegib and its potential in the treatment of advanced basal cell carcinoma
Muzafar A Macha1, Surinder K Batra, Apar Kishor Ganti
1Department of Biochemistry and Molecular Biology, University of Nebraska Medical Center, Omaha, NE, USA.
Abstract:
Basal cell carcinoma (BCC) is the most common human malignancy. Recent advances in our understanding of the critical biologic pathways implicated in the development and progression of BCC have led to the development of the first molecular targeted therapy for this disease. The hedgehog pathway is mutated in virtually all patients with BCC and recent trials with vismodegib, an inhibitor of this pathway, have shown significant responses. This review will discuss the importance of the hedgehog pathway in the pathogenesis of BCC and describe in detail the pharmacology of vismodegib in relation to its activity in advanced BCC.
Insights
Basal cell carcinoma (BCC), the most common human cancer, is now treatable with vismodegib, a targeted therapy. This drug inhibits the mutated hedgehog pathway crucial for BCC development, showing significant patient responses.
Area of Science:
- Oncology
- Dermatology
- Molecular Biology
Background:
- Basal cell carcinoma (BCC) is the most prevalent human malignancy.
- Understanding BCC's molecular pathogenesis has enabled targeted therapy development.
- The hedgehog signaling pathway is frequently altered in BCC.
Purpose of the Study:
- To review the role of the hedgehog pathway in BCC development.
- To detail the pharmacology and clinical activity of vismodegib.
- To discuss vismodegib as a targeted therapy for advanced BCC.
Main Methods:
- Literature review of hedgehog pathway in BCC.
- Analysis of clinical trial data for vismodegib.
- Pharmacological review of vismodegib's mechanism of action.
Main Results:
- Hedgehog pathway mutations are nearly universal in BCC.
- Vismodegib, a hedgehog pathway inhibitor, demonstrates significant efficacy.
- Targeted therapy offers a new treatment modality for advanced BCC.
Conclusions:
- The hedgehog pathway is a critical driver of BCC.
- Vismodegib represents a successful targeted therapy for advanced BCC.
- Further research into pathway-specific treatments is warranted.
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