The Hedgehog Signaling Pathway as a Therapeutic Target in Pediatric Liver Tumors

Recent evidence has indicated that Hedgehog signaling significantly contributes to liver development and regeneration. However, constitutive activation of the pathway drives growth of liver tumors, both in adults and children. This talk will focus on strategies set up to block the Hedgehog signaling cascade on multiple levels. It will describe the current options to toggle the pathway by inhibiting transmembrane proteins using antagonists, small-molecule inhibitors of downstream effectors, and compounds which strive against epigenetic alterations of pathway components. The presented approaches will illustrate the promise and challenges associated with pharmacological reagents that can block oncogenic Hedgehog pathway activity.