ARTICLE | Cover Story

Arsenic against the hedgehog

August 5, 2010 7:00 AM UTC

Researchers at the Stanford University School of Medicine have found that arsenic trioxide is able to antagonize the hedgehog pathway.1 The findings could open up hedgehog-driven solid tumors such as basal cell carcinoma and medulloblastoma as new indications for Cephalon Inc.'s Trisenox arsenic trioxide, which already is marketed for acute promyelocytic leukemia, a form of blood cancer.

Aberrant activation of the hedgehog (Hh) pathway can result from loss-of-function mutations in patched 1 (PTCH1), which is an inhibitory cell surface receptor in the pathway, through activating mutations in smoothened(SMO), which is a membrane component downstream of PTCH1, or in intracellular pathway components downstream of SMO.2 Although a handful of biotechs are attacking SMO, a team led by Philip Beachy, a professor of developmental biology at Stanford and a member of the university's Institute for Stem Cell Biology and Regenerative Medicine, has been searching for ways to block downstream targets...