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GDC-0449 reduced tumor size in basal cell carcinoma
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SAN DIEGO — GDC-0449, a first-in-class, oral systemic inhibitor of the Hedgehog signal transduction, has shown significant antitumor effects in a phase-1 study of nine patients with basal cell carcinoma, presented yesterday at the 2008 Annual Meeting of the American Association for Cancer Research.
Durable clinical benefit was seen in eight of the nine patients enrolled, according to Daniel D. Von Hoff, MD, physician in chief at Translational Genomics Research Institute in Scottsdale, Ariz.
“Medical oncologists might not see many patients with metastatic basal cell cancer in their career, but they do see them,” Von Hoff told HemOnc Today. “In our study, a patient with basal cell carcinoma was already treated with platinum and carboplatin and kept progressing, but we gave him this medication with virtually no side effects and he had a dramatic response.”
A dose escalation portion of the study enrolled one patient at 150 mg, one at 270 mg, and one at 540 mg of GDC-0449 assigned orally as a single dose. An additional six patients were enrolled at 150 mg GDC-0449 in an expansion cohort.
Eight patients have been evaluated; one patient died. Six patients had partial response: two radiologic partial responses and four partial responses by clinical exam. Two patients have stable disease and disease progressed in another patient. GDC-0449 had a total clinical benefit of 89%.
“Hedgehog is such an important pathway and appears to be turned on in so many other tumors,” Von Hoff said. “Inhibiting the Hedgehog pathway has potential for many other types of cancer.” – by Leah Lawrence
This is most interesting. We lack effective agents for metastatic basal cell carcinoma, and the relevance of this pathway to progression of this neoplasm is well documented. The other area of recent interest in the therapy of basal cell carcinoma relates to toll-like receptor agonists, so we are potentially poised for more rational combination therapy given the association of this neoplasm with immunodeficiency, as well.
– John M. Kirkwood, MD
Division of Hematology/Oncology
University of
Pittsburgh
For more information:
- Von Hoff DD. #LB-138. Presented at: 2008 Annual Meeting of the American Association for Cancer Research; April 12-16, 2008; San Diego.