Saturday, June 7, 2014

Nintedanib's Phase 3 Study in Plain English

As you know, I am most excited about the cancer drug Nintedanib's Phase 3 study in slowing fibrosis. But, sometimes the data is hard to plow through. On Thursday, I had tea with my friend Lois who was a drug designer. She has taught me a lot. I asked her to read the study and let me know what it means in plain English. Here is her reply:

 It turns out that nintedanib is NOT a monoclonal antibody, but a small molecule made by Boehringer Ingelheim as a cancer drug.  It doesn't kill cancer cells, so is not a traditional toxin.  Instead it binds to a family of growth receptors and inhibits angiogenesis or the formation of new blood vessels. Cancer cells need at lot of nutrients and oxygen to support their uncontrolled growth, so pump out factors that increase blood vessel formation as they grow.  If this process is inhibited, which nintedanib does, the cancer is starved of food and essentially dies of starvation.
  

I don't know to what extent your lung disease fibrosis extent is also caused by new blood vessel
formation or if the fibrosis itself can be inhibited by tyrosine kinase driven receptors.  Tyrosine
kinases are involved in all kinds of growth stimulation and work by phosphorylating other proteins
in a whole network of proteins within the cells.  It does make sense in the biology world that inhibiting an unwanted growth process would slow a disease.
  

The article itself sounds very positive in that the FVC declined by about 50% less in the treated group as compared with the placebo.  Some patients actually had declines of less than 5% over a year, which is somewhat miraculous.  The major adverse effects were diarrhea in about 60% of subjects and nausea in 20%.  It looks really, really good.  I would definitely talk with your lung goddess about trying to get yourself included in another clinical trial, if that is scheduled, or sign up for it once it gets approved.  After all there really is nothing else out there for this particular disease.

So, there it is in plain English. It is coming for those of us with fibrotic lung diseases. Hang on. Soon.

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