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Updated: Feb 10, 2026

BioMEMS and Cellular Biology: Perspectives and Applications
Published on: October 1, 2007
Vismodegib and the Hedgehog Pathway Inhibitors: A Historical Perspective to Current Clinical Application
Abstract:
Vismodegib (Erivedge, Genentech-Roche) is the first in class of Hedgehog pathway inhibitors approved for treatment of metastatic basal cell carcinoma (BCC), or locally advanced BCC that has recurred after surgery or is not amenable to surgery or radiation. Its path to discovery has been unique and traces its origin to corn lilies, sheep, Drosophila flies, and the Hedgehog signaling pathway. J Drugs Dermatol. 2018;17(5):506-508.
Insights
Vismodegib is a groundbreaking Hedgehog pathway inhibitor for advanced basal cell carcinoma (BCC). Its unique discovery journey highlights the importance of fundamental research in developing novel cancer therapies.
Area of Science:
- Oncology
- Molecular Biology
- Dermatology
Background:
- Basal cell carcinoma (BCC) is the most common human cancer.
- Advanced BCC often requires novel therapeutic approaches.
- The Hedgehog signaling pathway is crucial in embryonic development and implicated in BCC pathogenesis.
Observation:
- Vismodegib represents the first approved drug targeting the Hedgehog pathway.
- Its development involved interdisciplinary research, from natural sources to molecular targets.
- The drug is indicated for metastatic or locally advanced BCC unresponsive to surgery or radiation.
Findings:
- Vismodegib's approval marks a significant advancement in BCC treatment.
- The drug's efficacy in advanced BCC cases has been clinically validated.
- Its unique origin story underscores the value of diverse scientific exploration.
Implications:
- Vismodegib offers a new therapeutic option for patients with difficult-to-treat BCC.
- The success of Vismodegib may pave the way for other pathway-specific inhibitors.
- Understanding the Hedgehog pathway's role in cancer opens avenues for future drug discovery.
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