Biochemical Breakthrough - Fragile X Syndrome

Doctor Gul Dolen discusses the significance of finding a potential biochemical treatment for the neurological disorder, Fragile X syndrome.

It’s really a new thing in neuroscience. Neuroscience is obviously developing and I would say that our discovery is one of the first times that we’ve been able to treat a disease by understanding what causes the disease. Most psychiatric therapies in existence today were discovered somewhat accidentally. Here is a case where we decided to study Fragile X syndrome with a very specific theory in mind. We said we believe plasticity is disrupted, we predict that it will be disrupted and we predict that this abnormality in this one type of receptor is what’s causing all of the problems. We were able to make correlations between features of the disease, symptoms of the disease and predictions of what metabotropic glutamate receptors do and draw those parallels. By doing that we had a very strong hypothesis about what would happen when we treated the Fragile X knockout mice by reducing their metabotropic glutamate receptor signaling. So, it’s a therapy that is rooted in an understanding of the mechanisms of neuronal plasticity, and that’s really a first.