Cyclopamine

Cyclopamine

Cyclopamine is a naturally occurring chemical compound that belongs to the group of steroidal jerveratrum alkaloids. It is a teratogen isolated from the Corn lily (Veratrum californicum) that has been found to have inhibitory effects on the Sonic Hedgehog signaling pathway (SHH).

History

Cyclopamine was first identified in the 1960s when it was discovered that lambs born to ewes that had grazed on corn lily during pregnancy developed a condition known as cyclopia, a severe birth defect where the face is not properly divided into two halves. The compound was named after this condition.

Chemical Structure and Properties

Cyclopamine is a steroidal alkaloid with a complex structure that includes a fused five-ring system. It is a solid at room temperature and is soluble in organic solvents such as ethanol and DMSO. Its molecular formula is C32H41NO2.

Biological Activity

Cyclopamine acts by binding to and inhibiting the activity of Smoothened, a protein that plays a crucial role in the SHH pathway. This pathway is involved in the regulation of cell growth and differentiation, and its disruption has been implicated in a number of diseases, including various types of cancer.

Medical Applications

Due to its ability to inhibit the SHH pathway, cyclopamine has potential therapeutic applications in the treatment of certain types of cancer, including basal cell carcinoma and medulloblastoma. However, its use in humans is currently limited due to its toxicity and poor bioavailability.

Safety and Toxicity

Cyclopamine is highly toxic and can cause severe birth defects if ingested by pregnant animals. It is also toxic to cells in culture, and its use is therefore restricted to research settings.

See Also

• Corn lily
• Sonic Hedgehog signaling pathway
• Smoothened
• Cyclopia

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