Potential Reversal of Neurodevelopmental Disorders

Successful treatment of three mouse models of neurodevelopmental disorders:
Fragile X, Rett and Angelman syndromes

2007 saw the publication of several studies that documented successful treatment of disease symptoms in mouse models of three different neurodevelopmental disorders related to autism. Most significantly, two of three (Fragile X and Rett) involved reversal of the phenotype AFTER the mice had already become sick, suggesting that developmental disorders such as autism may be treatable in adolescence or adulthood.

In February, researchers in Scotland found that they could reverse the debilitating defects and certain death in mice carrying the Rett syndrome gene, well after the mice had regressed into the most severe stages of disease. In July, researchers at Massachusetts Institute of Technology used a mouse model of Fragile X syndrome to show that it was possible to reverse Fragile X and autistic symptoms after birth. Although the specific treatment approaches may not be directly applicable to an eventual autism treatment, the successful and entirely unexpected “rescue” of adult animals taught the world that so-called "developmental" disorders, those that begin in infancy, apparently still have a potential to be reversed later in life.

Given the behavioral overlap of these disorders with autism, even if the underlying disease-causing biological mechanisms are different, these results provided hope to scientists and families alike that we can (and will!) do the same for autism.