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Cyclopamine is a steroidal alkaloid which has been extracted from corn lily (Veratrum californiucum). It has shown potential as an anti-tumor agent and is undergoing Phase 1 trials for FDA Approval.5.

Click to see US Patent data for Cyclopamine.


  • Causes birth defects, including cyclopia, in animals when the plant is ingested by pregnant female.
  • Targets HedgeHog (HH) signaling pathway. Strict control is critical for correct cell differentiation during embryo genesis. In adults, HH signaling plays role in regulation of stem-cell maintenance and proliferation.
  • Misregulation of HH signaling has been shown to cause tumors, including basel cell carcinoma, medulloblastoma, small cell lung cancer, pancreatic adenocarcinoma, and some prostrate cancers.
  • HH signaling has also been shown to be important in the maintenance of breast, brain, and clorectal cancers. Shows very selective activity against HH-dependent tumors and is well tolerated. Able to target cancer stem cells that are linked to cancer reoccurrence.
  • Carbohydrate-Cyclopamine conjugates disclosed through US Patent. These compounds include pyranose, furanose, and mannitol conjugates, linked through a 1,2,3-triazine ring to the nitrogen of cyclopamine. Methods for preparing these compounds, pharmaceutical compositions containing these compounds, and their use as anticancer agents are also disclosed.3

Chemical Structure of Cyclopamine


Cyclopamine is partially soluble in, Methanol, Ethanol, DMSO and Benzene at room temperature, but will precipitate out at -20°C. Cyclopamine is freely soluble in Methylene Chloride and Chloroform.


1. Alam MM, Sohoni S, Kalainaykan SP, Garrossian M, Zhang L. Cyclopamine tartrate, an inhibitor of Hedgehog signaling, strongly interferes with mitochondrial function and suppresses aerobic respiration in lung cancer cells.. BMC Cancer. 2016 Feb 24;16(1):150. doi: 10.1186/s12885-016-2200-x. PMID: 26911235. http://www.ncbi.nlm.nih.gov/pubmed/26911235 (accessed Feb 28, 2016).
2. Borzillo Gary V., et. al. Current Topics in Medical Chemistry 5: 147-157 (2005).
3. Chang Cheng-Wei T., Garrossian Massoud. (Utah State University). Carbohydrate-Cyclopamine Conjugates as Anticancer Agents. US Patent Application #20110275576, Filed: January 6, 2010. Issued: November 10, 2011.
4. Chen J.K., et al. Genes & Development 16: 2743-2748 (2002).
5. Curis/Genetech. Skin Cream targeting basal cell melanoma.
6. Harper M. The curious case of the one eyed sheep, www.forbes.com/global/2005/1128/066A-print/html (2005).
7. Incardona J.P., et al. Development. 125: 3553-3562 (1998).
8. Katano M., et al. Cancer Letters. 227; 99-104 (2005).
9. Lee Stephen T., Welch Kevin D., Panter Kip E., Gardner Dale R, Garrossian Massoud, Chang Cheng-Wei Tom. Cyclopamine: From Cyclops Lambs to Cancer Treatment. American Chemical Society [Online] 2014, 10, 1021 http://pubs.acs.org/doi/abs/10.1021/jf5005622 (accessed Feb 28, 2016).
10. Qualtrough D., et al. International Journal of Cancer. 110 831-837 (2004).
11. Tabs S., Avci O. Eur J Dermatol. 14: 96-102 (2004).
12. Taipale J., et al. Nature 406 944-945 (2000).