Biochemical Treatments for Autism?

Doctor Gul Dolen discusses how new biochemical treatments for Fragile X Syndrome may be used to treat autism.

Right now, the status of the project is that we have proven in principle in the mouse model that in fact the protein missing in Fragile X, which is FMRP, and mGluR work in functional opposition. We did that in mice and we used genetics to do it because when you use molecular manipulations to turn down signaling, it’s very clean; you know exactly what you’re doing, you know exactly how much of the protein is available and it’s very precise. But in humans of course you wouldn’t be able to do that sort of manipulation, at least not yet. So the way that we are approaching this issue in humans is pharmacologically. Indeed, based on our findings, clinical trials are underway in humans. A number of companies are developing metabotropic glutamate receptors as therapeutic targets for Fragile X. Really, what’s very exciting is that by looking at this pathway and figuring out how synaptic plasticity is disrupted, and understanding how metabotropic signaling works, it’s clear that mGluRs and FMRP don’t exist in isolation at the synapse and that there are a number of other proteins that interact with these proteins, and they are all found in the synapse. Interestingly a number of the proteins that interact with mGluRs have also been identified as candidate genes for autism. Those candidate genes interact with mGluRs and it suggests that the therapies that we’ve developed for Fragile X may be effective, although that is not known at this point. But it’s very exciting because, at least theoretically, it may be that the drugs that we develop to cure Fragile X will be useful in some, if not all, causes of autism.