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

Abstract

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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