Disrupting Hedgehog May Reverse Advanced Cancer, If Only Temporarily

摘要

In the first clinical proof of its kind, a drug has dramatically shrunk cancerous tumors by disrupting a key genetic pathway. But a study targeting one deadly brain cancer, medulloblas-toma, ended in disappointment as the patient's once-tamed tumor quickly developed resistance to the drug and killed him.rnThe drug, GDC-0449, was developed at Genentech in San Francisco, California. It locks onto and deactivates a protein, Smoothened, that activates the Hedgehog signaling pathway, which in turn orchestrates rnhow embryonic stem cells develop. In adults, the Hedgehog pathway is dormant, but it awakens in many cancers.