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Virus-induced Gene Silencing (VIGS) in Nicotiana benthamiana and Tomato

Virus-induced Gene Silencing (VIGS) in Nicotiana benthamiana and Tomato

Published on: June 10, 2009

Vismodegib.

Charles M Rudin¹

¹The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, Maryland 21287, USA. rudin@jhmi.edu

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research

|June 9, 2012

Summary

This summary is machine-generated.

Vismodegib is a new oral medication approved for advanced basal cell carcinoma. It showed significant response rates in patients ineligible for surgery or radiation.

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Area of Science:

Oncology
Pharmacology

Background:

Basal cell carcinoma (BCC) is the most common human cancer.
Advanced BCC (metastatic or locally advanced) lacks effective treatments, especially for patients unsuitable for surgery or radiation.
Vismodegib (GDC-0449) targets the Hedgehog signaling pathway, crucial in BCC development.

Purpose of the Study:

To review the mechanism of action, preclinical data, and clinical trial results of vismodegib.
To discuss the U.S. FDA approval of vismodegib for advanced BCC.
To explore the potential of vismodegib in other disease contexts.

Main Methods:

Review of preclinical studies on vismodegib's efficacy and mechanism.
Analysis of a nonrandomized, parallel cohort Phase II study in 99 advanced BCC patients.
Evaluation of objective response rate (ORR) and progression-free survival (PFS) as primary endpoints.

Main Results:

Vismodegib demonstrated objective response rates of 30.3% in metastatic BCC and 42.9% in locally advanced BCC.
Median progression-free survival was 9.5 months for both patient cohorts.
The drug's approval was based on these outcomes, addressing a significant unmet need.

Conclusions:

Vismodegib represents a breakthrough therapy for advanced basal cell carcinoma.
Its efficacy in clinical trials supports its role in patients with limited treatment options.
Further research is ongoing to evaluate vismodegib's potential in other cancers and in combination therapies.