Science-- there's something for everyone

Wednesday, May 26, 2010

Possible drug treatment for one type of autism

Before I report on this story, I’d like to caution my readers that this treatment is only in the earliest stages of testing. Still, it’s pretty exciting news. Joseph Buxbaum of the Seaver Autism Center for Research and Treatment at Mount Sinai School of Medicine and his colleagues have successfully improved nerve cell connections in mice by giving them a derivative of insulin-like growth factor-1 (IGF1).

The mice in question have a gene mutation called SHANK3, which is associated with Phelan-McDermid Syndrome (an autism spectrum disorder). In humans, along with physical abnormalities, the SHANK3 mutation causes language delays and other autistic symptoms. Mice with the same mutation have nerve cells that cannot communicate with each other properly, leading to neurological symptoms.

As an aside, Gudrun Rappold and his team from Heidelberg University Hospital have now implicated the SHANK2 gene in autism as well. The Shank proteins are molecular scaffolds, meaning they function as support structures that hold other proteins or factors in their proper places. They appear to be critical to the structure and function of nerve synapses.

But back to our SHANK3 mouse studies. Buxbaum and his team treated the mice with IGF1. In only two weeks, the mice had improved nerve cell communication and were able to react normally to stimuli. The hope is that by restoring nerve function, autism spectrum symptoms would be diminished or eliminated. The scientists plan to continue observing the mice to determine what specific neuronal changes were caused by the IGF1 injections.

As I said, this treatment is not yet ready for human trials. However, these preliminary tests are promising, especially since IGF1 is already approved for human use (to treat growth failure), and thus is likely to be relatively safe.