Research Wednesday

Back to our normal grind here with some more promising new medication to help out FXS kiddos (and adults).  This one is a little different than our clinical trial drugs though as the medication I am covering today is already FDA approved!

Many of you may have heard of minocycline, a drug used to treat bacterial infections including acne.  If you haven't heard of minocycline, you most likely will have heard of tetracycline (though maybe not by name). Tetracycline was used for a while as a "safe" antibiotic for pregnant women but was later found to permanently stain the baby's teeth.

So, how does Minocycline connect with FXS?  Many families whose children are on minocycline are showing improvements in behavior, attention levels, and most importantly language!

It all started with a mouse.  See, a fragile x knockout mouse was given minocycline for three weeks starting at birth and researchers found that the synaptic connections were normalized.   Normally in FXS, those synaptic connections are weak which is thought to be the cause of intellectual disability and autism.  The minocycline treatment in the mouse reversed that and also showed good effects on cognition and learning tasks as well.   After the study was published, many parents wanted their FXS children to try minocycline and a good amount of doctors decided to prescribe it to see what happened.

The amount of FXSers on this, prompted a survey to parents in which a whopping 54% were reporting improvements in language, 50% reported improved attention span, 44% noticed improved social communication, and 30% noticed a lessening of anxiety levels. 

All these promising survey results resulted in a controlled study of minocycline in FXS patients and is currently underway.  I've spoken to many parents whose children are on minocycline and showed improvement very quickly (within a few weeks, actually).  As with any medicine, there are those FXSers who don't respond as well as others, but from the studies and reports I have heard those numbers are fairly small.

I think the most promising thing to me is seeing that there are medications out there now that are proving to treat some of the symptoms of FXS, and that is excellent to hear.