Modulators of hedgehog Signaling

Members of the Hegdehog (Hh) family of secreted signaling proteins play critical roles in coordinating cell growth and differentiation. Impaired pathway function in embryogenesis can produce severe malformations, such as cyclopia and other midline defects of the face and forebrain associated with deficits in Sonic hedgehog (Shh) signaling. In contrast, constitutive activation of the Hh signaling pathway after completion of embryogenesis is associated with the formation of tumors. This invention consists of a series of small chemical compounds that modulate Hh signaling without high levels of general cytotoxicity Description (Set) Proposed Use (Set) The compounds may be useful for the treatment of disorders resulting from inappropriate activation of the Hedgehog signaling pathway. These disorders include cancers such as basal cell carcinoma (BCC), the most common cause of cancer in Caucasians with 750,000 new cases annually in the U.S. alone, primitive neuroectodermal tumors (PNETs) including the most common childhood brain tumor, medulloblastoma, as well as rhabdomysarcoma, and fibrosarcoma. These compounds also serve as useful starting points for the further synthesis of even more potent pathway modulators. Patent (Set) WO 03/088970

Patents:
US 3,088,970

Inventor(s): Beachy, Philip A.

Type of Offer: Licensing



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