Cyclopamine

Cyclopamine (11-deoxojervine) is naturally-occurring chemical that belongs to the group of steroidal jerveratrum alkaloids. It is a teratogen isolated from the corn lily (Veratrum californicum) that can lead to cyclopia (holoprosencephaly).

Cyclopamine inhibits the hedgehog signaling pathway (Hh) by influencing the balance between the active and inactive forms of the Smoothened protein. Cyclopamine is currently being investigated as a treatment agent in basal cell carcinoma, medulloblastoma, and rhabdomyosarcoma, tumors that result from excessive Hh activity, glioblastoma, and as a treatment agent for multiple myeloma.

Cyclopamine was named for one-eyed lambs which were born to sheep which grazed on wild corn lily at a farm in Idaho. In 1957 the US Department of Agriculture started an eleven year investigation which led to the identification of cyclopamine as the cause of the birth defect.