Return of rain mouse

In 2014, I reported a remarkable find in The Economist where researchers used a sleeping sickness drug to treat mice suffering from the rodent equivalent of autism. Administration of the drug, known as suramin, caused the autism traits to fade away over a matter of weeks. Similarly, as the drug treatment ended, the autistic traits came back. It was astonishing stuff but my editor and I were cautious in our reporting since what works in mice often does not work in men. Well, humans trials have now finished and the findings suggest that suramin has tremendous potential.

What works in mice is now working in men.  Image courtesy of Aaron Logan.

What works in mice is now working in men.

Image courtesy of Aaron Logan.

Like all first trials in humans, this one was small with just ten patients. All were autistic boys. Half were given a placebo and half were given suramin. The participants went through numerous tests throughout the treatment to monitor their behaviours and tease out whether the grip that autism had on them was weakened. All five of the kids who were given the suramin showed substantial gains while the drug was given while none of the children given the placebo did.

Obviously, this is a big deal since there is currently no drug available for treating autism but what these findings suggest from a biochemical perspective is perhaps even more important. Suramin gums up purine receptors on cells and prevents neurons (cells in the brain) in particular from entering what is known as the cellular danger response. While in this defensive mode, neurons become more resistant to a wide range of diseases but stop making connections with other neurons. This is not really a problem when someone is ill for a few days or weeks but the theory with autism is that patients with the condition have neurons that permanently get stuck in the danger response and can't stop responding to purine stimulation. The fact that suramin improves language and social skills in autistic patients while simultaneously decreasing repetitive behaviours suggests that the theory of the cellular danger response being a central cause of autism is likely correct.  

This work has to be replicated before we can take it too seriously but the fact that what works in mice is working in autism sufferers is really something extraordinary. You can read more in The Economist article that I wrote on this here.