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Updated: May 13, 2026

Cell Population Analyses During Skin Carcinogenesis
Published on: August 21, 2013
Advanced basal cell carcinoma of the skin: targeting the hedgehog pathway
Aleksandar Sekulic1, Aaron R Mangold, Donald W Northfelt
1Department of Dermatology, Mayo Clinic, Scottsdale, Arizona, USA. sekulic.aleksandar@mayo.edu
Purpose Of Review:
This article provides an update on basal cell carcinoma (BCC), with a focus on the advanced BCC (aBCC), and the recent progress with targeted hedgehog signaling pathway inhibition for treatment of aBCC.
Recent Findings:
The hedgehog signaling pathway is aberrantly activated in most BCC tumors providing an attractive therapeutic target in this cancer. Recently developed targeted hedgehog pathway inhibitors have demonstrated remarkable efficacy in the treatment of aBCC and the first oral hedgehog pathway inhibitor vismodegib was granted the US Food and Drug Administration (FDA) approval. Toxicities of the current hedgehog pathway inhibitors are mostly mild to moderate, but with prolonged treatment can pose a therapeutic challenge.
Summary:
Hedgehog pathway inhibition is a novel and powerful approach for treatment of aBCC. Current research efforts aim to enhance the activity and minimize toxicity of this promising new therapy.
Insights
Targeted hedgehog pathway inhibitors show significant efficacy in treating advanced basal cell carcinoma (aBCC). Research is ongoing to improve treatment activity and reduce side effects for this novel therapy.
Area of Science:
- Oncology
- Dermatology
- Molecular Biology
Background:
- Basal cell carcinoma (BCC) is a common skin cancer.
- Advanced BCC (aBCC) presents unique treatment challenges.
- The hedgehog signaling pathway is frequently dysregulated in BCC.
Purpose of the Study:
- To provide an update on basal cell carcinoma (BCC).
- To focus on advanced BCC (aBCC) treatment.
- To review progress in targeted hedgehog signaling pathway inhibition for aBCC.
Main Methods:
- Review of recent scientific literature on hedgehog pathway inhibitors.
- Analysis of clinical trial data for targeted therapies in aBCC.
- Evaluation of the efficacy and toxicity of hedgehog pathway inhibitors.
Main Results:
- Aberrant hedgehog signaling pathway activation is a hallmark of BCC.
- Targeted hedgehog pathway inhibitors demonstrate remarkable efficacy in aBCC.
- Vismodegib, the first oral hedgehog pathway inhibitor, received FDA approval.
- Current inhibitors present mild to moderate toxicities, posing challenges with prolonged use.
Conclusions:
- Hedgehog pathway inhibition represents a novel and potent therapeutic strategy for aBCC.
- Ongoing research aims to optimize the efficacy and safety profile of these inhibitors.
- Further development is crucial for managing advanced basal cell carcinoma effectively.
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