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A Mouse is not a Human Chapter 5.2X10^23

5/29/2013

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Early last year, it was reported that Bexarotene (a Ligand product that was discovered when I worked there) cleared amyloid plaques from mice, prompting the hypothesis that the retinoid X receptors were somehow involved in Alzheimer’s disease.  I have to admit I have a soft spot for nuclear receptors in general and rexinoids in specific, having worked on them for so long.  However, this was mouse data and as I have pointed out here and in other places, a mouse is not a human.  So it wasn’t really that surprising that several research groups reported today that they can’t reproduce the experiments. Oh Snap! 

This would fall in to the category of things I find mildly disappointing, except for the fact that doctors have apparently prescribing Bexarotene to their Alzheimer’s patients off label.  Having been at Ligand for a good portion of its troubled history, I know that Bexarotene has some pretty nasty side effects.  Hyperlipidemia that is sufficiently elevated to cause pancreatitis.   I have very serious reservations about off label prescriptions, particularly based on one study.  I know that Alzheimer’s is a horrible disease, but remember our friend Hippocrates, first do no harm.  This clearly falls into the doing harm part of the equation.  

Let’s remember A MOUSE IS NOT A HUMAN.

Sorry for shouting. 


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    Adam Kallel Ph. D.

    Our CSO sounds off about drug discovery, computational chemistry and history

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