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


OBJECTIVE
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.
Doggrell
Sheila A
SA
Queensland University of Technology, School of Life Sciences, Faculty of Science and Technology, GPO Box 2434, QLD4001, Brisbane, Australia. sheila.doggrell@qut.edu.au