Breakthrough: Autism Studies Point to Cause/Treatments

This recent astonishing study (summary) showed that mice genetically engineered to have autism were successfully treated by an application of neuronal nitric oxide synthase (nNOS) inhibitor, 7-nitroindazole (7-NI or S-nitroso-N-acetyl penicillamine), for 10 consecutive days. They tested two types of mice with different autism-inducing mutations. In one type treatment reversed autism symptoms in both sexes, the other reversed them in males.

This study shows not only that oxidative stress from N2O contributes to autism pathology, but that there is another compound (NDGA) which helps the body convert N2O into the non-deleterious form also effectively treats autistic symptoms.

Here is yet a third study that shows yet another N2O inhibitor (catechin hydrate)(or convertor to another form) successfully treats autistic symptoms in rodents.

Indeed, even curcumin seems to have some positive effect, though I suspect it is mopping up the damage after the excess N2O is created rather than cutting of the problem at its core. Also, the study was done on rats where autism-like symptoms were artificially induced whereas the other studies also used rodents genetically modified to have autism. Just as in the Revesterol study, which also had positive results for the same reason. I don’t say these two will be effective as they are general, not nervous-system targeted, and deal with mopping up the damaging excess N2O after it has been present.

You may say “but that was with rodents”. It turns out they have understood the connection for years. Here is a human study from 2013 where children were given a compound (sapropterin) that was more broadly targeted but also impacted N2O. It is an anti-fungal too I believe, so it may be promising for more than one reason. It showed significant improvement on some but not all of the spectrum. And they suspected even then that it was the effect on NO that was making the difference. They also did a human study with the closely related Tetrahydrobiopterin and found that it did not improve everything but did improve social awareness, autism mannerisms, hyperactivity, and inappropriate speech. This was ten years ago.

I suspect that if their brain had years to heal and repair, they would see improvements in the other areas too. Take a brain that has been battered by long-term immune response, just because you remove the source of the abuse doesn't mean the brain heals right away.

The epilepsy/bipolar drug I had noticed before was effective if the cause of autism is a mutation in a particular gene, but the N2O theory of autism would apply to a wide variety of stem issues. There are lots of ways the N2O balance could be off, and restoring the balance would treat a wide variety of things that could lead to the same issue.

The body needs N2O. So using a general inhibitor over a long time without monitoring would be too risky. Inhibition interventions are best when targeted and there is monitoring of levels from time to time. But leaving the nervous system with all of this excess is a known problem.