A potential new investigational therapy for advanced and metastatic basal cell skin cancer is revealed by researchers.
The trial, sponsored by Genentech, included clinicians at TCRS, the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University in Baltimore, and Karmanos Cancer Institute in Detroit.
The results demonstrated that GDC-0449, a Hedgehog Pathway Inhibitor, appears to shrink tumors in locally advanced and metastatic BCC. Known as the “Hedgehog” trial, the clinicians observed a durable clinical benefit -defined as tumor shrinkage visible on X-ray or other physical exam or improvement in symptoms without tumor growth- in 18 of 33 patients evaluated. Others had stable disease for prolonged periods of time. Only 4 patients had progression of disease.
Abnormal activation of the Hedgehog signaling pathway appears to be an important mechanism for numerous types of cancer to develop, survive, or grow. A chemical called cyclopamine found in the California corn lily can inactivate this Hedgehog pathway.
Study investigators selected BCC as the first cancer to study in that most BCCs have abnormalities or mutations of Hedgehog pathway genes named PATCHED and SMOOTHENED.
“Success of this new therapy is another example of applying genetic information to medicine. We are constantly working to improve treatment options for patients with common and rare cancers,” said TCRS physician Glen J. Weiss, M.D., a contributing author on the study.
Patient response to the therapy was assessed through physical examination and imaging.
“Integrating genomic data with state-of-the-art clinical and imaging information to develop and apply targeted therapies has certainly taken a major step forward with the encouraging results from the Hedgehog trial,” added Dr. Ron Korn, a Scottsdale Healthcare radiologist and director of Scottsdale Medical Imaging Ltd.
The study, conducted at TGen Clinical Research Service (TCRS) at Scottsdale Healthcare and two other sites appears to demonstrate tumor shrinkage and limited side effects.
Source: The Translational Genomics Research Institute, USA