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Vismodegib in basal cell carcinoma

R N Amaria1, D W Bowles, K D Lewis

  • 1Cutaneous Malignancies and Developmental Therapeutics Programs, University of Colorado Cancer Center, Aurora, Colorado 80045, USA.

Drugs of Today (Barcelona, Spain : 1998)
|July 31, 2012
PubMed

Insights

Vismodegib effectively targets the hedgehog pathway, showing significant antitumor activity in preclinical and clinical studies for advanced basal cell carcinoma. This drug is now FDA-approved for treating unresectable or metastatic skin cancers.

Area of Science:

  • Oncology
  • Dermatology
  • Molecular Biology

Background:

  • Hedgehog signaling pathway activation is crucial in basal cell carcinoma (BCC) development.
  • Vismodegib is a novel small-molecule inhibitor targeting smoothened (SMO), a key component of this pathway.
  • Aberrant hedgehog signaling is implicated in various other tumor types.

Purpose of the Study:

  • To evaluate the efficacy and safety of vismodegib in preclinical models and clinical trials for BCC.
  • To establish vismodegib as a targeted therapy for advanced basal cell carcinomas.

Main Methods:

  • Preclinical studies in animal models assessing antitumor activity.
  • Phase I and II clinical trials in patients with advanced basal cell carcinomas.
  • FDA review and approval process for a novel cancer therapeutic.

Main Results:

  • Vismodegib demonstrated potent antitumor activity in hedgehog-dependent tumors, especially BCCs, in preclinical settings.
  • Clinical studies revealed dramatic anticancer responses in patients with advanced BCC.
  • FDA approval in January 2012 for specific indications.

Conclusions:

  • Vismodegib represents a significant advancement in targeted therapy for basal cell carcinoma.
  • The drug offers a new treatment option for patients with unresectable or metastatic BCC.
  • Targeting the hedgehog pathway is a validated strategy for treating certain skin cancers.

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