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Is Mystery Drug, Cyclopamine, Potential Multiple Myeloma Cure?

Home/Is Mystery Drug, Cyclopamine, Potential Multiple Myeloma Cure?

Is Mystery Drug, Cyclopamine, Potential Multiple Myeloma Cure?

A regular reader contacted me several weeks ago, asking about a drug called cyclopamine.  I had never heard of the drug, but I promised to do some research about it.

In his article titled Hedgehog Drugs Begin To Show Results, Ken Garber writes about what used to be called cyclopamine:

When researchers announced last month that a hedgehog pathway inhibitor from Genentech had shown activity in a phase I cancer clinical trial, few noticed. Phase I results are usually suggestive at best. But in this trial, eight of nine patients with advanced basal cell carcinoma responded to the Genentech drug, and all eight are still enrolled 16 months into the study. “The first patient in the world gets treated with an inhibitor of this pathway and has a dramatic response,” noted principal investigator Daniel Von Hoff, M.D., of the Translational Genomics Research Institute in Phoenix, Ariz., at the annual meeting of the American Association for Cancer Research. “That … is the essence of translational medicine.”

Best I can tell, the theory behind all of this hinges upon something called “hedgehog pathways.”  Here are a few more excerpts from the article:

Beginning in the late 1990s, Curran and others began trying to target hedgehog to treat these tumors with the help of transgenic mouse models. A natural-product hedgehog pathway inhibitor called cyclopamine pointed the way to new drugs…

…Now big pharmaceutical companies, including AstraZeneca and Bristol-Myers Squibb, are in on the hunt because these drugs might work for more common tumors. Beginning in 2002, researchers implicated hedgehog signaling in a variety of tumor types, including prostate, lung, pancreatic, stomach, and bladder cancers, as well as melanoma, glioblastoma, and multiple myeloma.

This reader and fellow patient is very serious about all of this.  Apparently, cyclopamine is a natural occuring compound which can be purchased openly without FDA approval.

Here is the link to this Oxford University Press 2008 journal article, along with additional links, Scottsdale Healthcare-TGen clinical trial results signal advances against skin cancer and Brain Tumor Stem Cells Killed By Experimental Anti-Cancer Drug.

The TGen article was published in 2009 and the Stem cell article in 2007. 

I feel like I know less about cyclopamine than when I started.  Are any of you familiar with it?  I will try to find out more, but at least this is a start.

Feel good and keep smiling!  Pat