Lessons from the Autistic Mouse

 

One surprising observation from reading the research on autism is how many times I have come across scientists making a mouse autistic and then showing how this can be reversed.

The important point is that the things the scientists did to the mouse (or its mother) are generally totally unrelated.  The only thing in common is the resulting mouse has “autistic behaviour”.

We can conclude that “autism” can be caused by entirely different events/disorders;  just like a head ache can be.

It would be correct to think of autism as a symptom, not a disease.

Perhaps, put a little clearer:-

• Autism is just a symptom of an underlying neurological disorder.  There are numerous such disorders, each with their own underlying pathology; some of these pathologies may overlap.  Treating the underlying pathology will moderate the symptoms of autism.

• Do not treat autism.  Treat the underlying pathology.

• A treatment that works in one person, with the symptoms of autism, may be completely ineffective in another.

• All treatments that are genuinely effective, in even the smallest group of children, should be carefully documented and shared publicly. Steps should be taken to look for biomarkers associated with each group.   

So, it is fundamental to think of autism as a symptom rather than a disease.

If researchers think of autism as a disease, it will likely remain incurable.  Granted, there is much more talk about autisms, sub-types and phenotypes, but this all goes out the window, so to speak, when it comes to doing clinical trials.

I am reading about a series of autism trials by Forest Laboratories of the US, using an old drug called Memantine.    I was shocked to see that they are trialing this drug in 118 locations around the world, on 906 children

An Open-Label Safety Study of Memantine in Pediatric Patients with Autism, Asperger's Disorder, or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS) 

Autism trials are usually tiny.  Conducting a trial on nearly a thousand kids is very expensive.  You would not undertake this kind of expense, unless you had a pretty good reason to think the drug was effective.

This trial is actually a safety study, in parallel there are a whole series of other studies.

Unfortunately, by trialing the drug on almost any kid, with any kind of autistic feature (Autism, Asperger’s, PDD-NOS), the important lesson from the mice has been completely lost.

While I really wish Forest Laboratories success, I suspect their trial will show that while Memantine is safe, it is not effective.

Over the years, there have been many anecdotal reports of the effectiveness of Memantine in some cases of autism.  Memantine is a drug that acts on the glutamatergic system by blocking NMDA receptors.  This will be explained in some detail in the next post “Ketamine, Memantine, D-Cycloserine and even Magnesium as Glutamatergic Modulators in Autism”.

Conclusion

You might think that much of the above should be common sense; sadly it is not.