Leper known

May 30, 2019

The story begins in the 1950s when thalidomide was approved as an over the counter sedative in Europe, but conspicuously rejected by the FDA in the US.  Shortly thereafter, it was discovered that thousands of babies were born without limbs or shortened limbs, a condition known as phocomelia. The mothers had all taken thalidomide during pregnanCcy.  It was an absolute tragedy, but also a major triumph for the United States FDA, which is still trumpeted to this day as a cautionary tale for rushing to approve drugs.

Thalidomide became the poster child for dangerous drugs and was untouchable for decades after in the Western world. However, it was discovered in the third world that this drug had a unique beneficial effect on incurable leprosy. Despite its reputation, it was approved as such in South American countries afflicted by the disease.

 

The drug remained ignored in the US however since there were so few cases of leprosy.  However, In the 1990s the legendary researcher Judah Folkman brought it back to life. Dr. Folkman was a surgeon at Harvard who commonly observed that cancer metastases flourished after he removed the primary tumor in a patient.  He deduced that the primary tumor was secreting something that suppressed the metastases. His research discovered that the factors responsible were inhibiting angiogenesis, or the growth of blood vessels into the tumor, which pioneered a major field in cancer therapy.  However, in those early days, there were no anti-angiogenic drugs.

 

His group looked for them and found one in the unlikeliest of places: thalidomide. It made perfect sense that phocomelia resulted from inhibition of blood vessel growth in the limbs and indeed Folkman’s group showed that thalidomide was anti-angiogenic.

 

That’s where Celgene comes in.  A small company with need for a starter product with significant upside.  They cleverly filed to approve thalidomide for leprosy, with an attendant program to screen for pregnancy.  They weren’t expecting many leprosy prescriptions, but the rarity of the disease and the lack of drugs for it along with the knowledge of how to mitigate thalidomides teratogenicity let the FDA to approve it.  Celgene knew cancer physicians would test it and, lo and behold, it wasn’t long before its activity in multiple myeloma was established and it was approved for that too. But it was already making millions of dollars off-label by the time that genuine approval rolled in.  From there, it was one thalidomide derivative and a few acquisitions on the way to a billion dollar market cap. 

 

Who says drug repurposing is pie in the sky?  Not us!  To write your own novel use of a drug success story, check out what Historeceptomics can do.

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