A new study conducted by researchers at Columbia University Medical Center (CUMC) reveals that the slowdown of normal brain "pruning" process during development was responsible for the surplus of synapses in the brain in autistic children and adolescents, which in turn has a big influence on how the brain functions as neurons use the synapses to connect and communicate with each other.
Although, the drug, rapamycin, has side effects that might preclude its use in people with autism. It was established that a drug that restored normal synaptic pruning could improve autistic-like behaviors in mice even when the drug was given after the behaviors have appeared.
AdvertisementUsing mouse models of autism, the researchers traced the pruning defect to a protein called mTOR. When mTOR was overactive, they found, brain cells lose much of their "self-eating" ability. And without this ability, the brains of the mice were pruned poorly and contained excess synapses.
The researchers could restore normal autophagy and synaptic pruning and reverse autistic-like behaviors in the mice, by administering rapamycin, a drug that inhibits mTOR. The drug was effective even when administered to the mice after they developed the behaviors, suggesting that such an approach might be used to treat patients even after the disorder has been diagnosed.
Alan Packer, PhD, said that the current view was that autism was heterogeneous, with potentially hundreds of genes that could contribute but that's a very wide spectrum, so the goal now would be now to understand how those hundreds of genes cluster together into a smaller number of pathways; that would give them better clues to potential treatments.
The study is published in the journal Neuron.