Anti Galectin 3 -- not actually a story from Star Trek

Interesting news from a clinical trial about NASH.  There is a lot of research going on for a way to treat or prevent cirrhosis.  Millions of us are headed to a bad end from liver disease but treatments have been elusive despite massive efforts.  It is still early but we may be seeing that hoped for light signaling success.

Galectin Therapeutics just released the result of a phase 2 clinical trial of a drug aimed at cirrhosis which reports positive effects.  It is the first trial result directly targeting cirrhosis that is likely to proceed to phase 3 trials and could be the first treatment to come out of the pipeline in the next few years.  They have a lot of work to do yet but the results today are encouraging.  They have demonstrated the ability to reduce the development of esophageal varices resulting from the increasing portal vein pressure resulting from advancing fibrosis.

WOW, too confusing. A little background perhaps. As damage becomes worse in the liver the pressure in the veins sending blood into the liver increases.  The veins of the esophagus, the throat, are connected and they are weaker so more pressure means those veins bulge like a balloon and are called varices.  Varices are a clear indication of serious liver disease so they indirectly measure overall liver blood flow.  If you do something which results in fewer varices you know that you have affected the liver even if you don't know exactly what you did.

OK, but anti galectin 3 seems kind of weird.  Well let me splain you.  In our body we have a family of 14 related proteins which are called galectins.  The chemistry of proteins is really complicated but they are everywhere in your body and are involved in a vast number of functions.  It turns out that galectin number 3 is associated with several not great things like cancer, inflammation, fibrosis, heart disease and stroke.  So a drug that blocks galectin 3 would be called an "anti galectin 3" and that is the kind of molecule used in this study.  Easy peasy if you say it quick.

This result is important because, even if the exact cellular activity is still under study, we have a molecule that has so far been shown to be safe and that has at least slowed the development of fibrosis in a year long very professional clinical trial.  There are a couple of other drugs advancing through trials at a similar pace but this is the first that works in this way and shows real promise as a drug that might be useful to stop fibrosis before it reaches serious levels.

This isn't a therapy, but it is a good step forward and we can hope that phase 3 trials leading to a useful treatment begin soon.  Also, remember that this is just one of hundreds of molecules being evaluated.  One of the biggest roadblocks may turn out to be a shortage of people willing to participate in clinical trials.  We are partnering with Antidote to provide a clinical trial finder on our website that you can use if you are interested in being involved.  We urge you to consider being a trial  participant as that is the only way that new treatments can come to clinical use.

A small bit of history, but I tried to get into this trial last year but I didn't pass the qualification tests so they didn't take me.  People tend to be afraid to participate in a trial but actually having really close medical monitoring is a good thing and trial participants generally have fewer problems than those who stay on the sidelines.  Just sayin.

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