The hedgehog pathway inhibitor GDC-0449 shows potential in skin and other cancers.

Abstract

BACKGROUND:

The hedgehog signalling pathway is vital in early development, but then becomes dormant, except in some cancer tumours. Hedgehog inhibitors are being developed for potential use in cancer.

OBJECTIVES/METHODS:

The objective of this evaluation is to review the initial clinical studies of the hedgehog inhibitor, GDC-0449, in subjects with cancer.

RESULTS:

Phase I trials have shown that GDC-0449 has benefits in subjects with metastatic or locally advanced basal-cell carcinoma and in one subject with medulloblastoma. GDC-0449 was well tolerated.

CONCLUSIONS:

Long-term efficacy and safety studies of GDC-0449 in these conditions and other solid cancers are now underway. These clinical trials with GDC-0449, and trials with other hedgehog inhibitors, will reveal whether it is beneficial and safe to inhibit the hedgehog pathway in a wide range of solid tumours or not.

• Authors: Sheila A Doggrell
• Journal: Expert opinion on investigational drugs (Expert Opin Investig Drugs) Vol. 19 Issue 3 Pg. 451-4 (Mar 2010) ISSN: 1744-7658 [Electronic] England
• PMID: 20078247 (Publication Type: Journal Article)