Suramin and the Fragile X (Fmr1 knockout) mouse model (and autism)

Fancy some weekend reading? Well, you could do a lot worse than having a gander through the paper by Jane Naviaux and colleagues [1] (open-access) discussing the results of a whole host of analyses following the use of the antipurinergic agent suramin on a mouse model of Fragile X syndrome.

Overprotective mother, forbidden road trip...

Regular readers might remember some previous discussions about suramin - a pharmaceutic designed to treat African sleeping sickness - and autism which have graced this blog (see here and see here). Following a series of studies which looked at the physiological and behavioural effects of suramin administration on a mouse model trying to recreate conditions of maternal immune activation (MIA (which itself has some autism research history), authors this time turned their attention to a mouse model of Fragile X syndrome, a condition which can in humans manifest with autistic traits (sometimes).

The Naviaux paper is a whopper in terms of data accumulated and results so I'm not going to even try and summarise the findings aside from quoting the authors that their: "results support the novel conclusion that antipurinergic therapy is operating by a mechanism that lies close to the root cause of the core behaviors and development in both the environmental MIA, and the genetic Fragile X models of ASD [autism spectrum disorder]. This mechanism appears to be traceable to mitochondria and regulated by purinergic signaling." Both mitochondrial and purinergic issues have featured in the autism research historical tapestry before (see here and see here respectively).

Just before anyone makes a run on suramin, I might however point out a few things: (a) the current and previous results are based on mouse studies and mice are mice not humans, and (b) suramin, whilst indicated for sleeping sickness, is not without the possibility of some pretty important side-effects (see here).

Still, this latest paper again potentially opens up a number of promising lines of inquiry in need of further investigation. And the added bonus is to see some more metabolomics included in their results!

To close: INXS and Mystify.

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[1] Naviaux JC. et al. Antipurinergic therapy corrects the autism-like features in the fragile X (Fmr1 knockout) mouse model. Molecular Autism 2015, 6:1

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Jane C Naviaux, Lin Wang, Kefeng Li, A Taylor Bright, William A Alaynick, Kenneth R Williams, Susan B Powell, & Robert K Naviaux (2015). Antipurinergic therapy corrects the autism-like features in the fragile X (Fmr1 knockout) mouse model Molecular Autism : 1186/2040-2392-6-1