Scientists from the Hector Institute for Translational Brain Research (Germany) have found that the drug lamotrigine, used to treat epilepsy and bipolar disorder, can alleviate the course of autism. The research was published in the journal Molecular Psychiatry.
Behavioral disorders seen in autism are associated with various genetic changes. In a new study, scientists discovered another molecular cause of this condition – “turning off” the MYT1L protein. Normally, it controls the activity of several genes in nerve cells. Mutations in the gene encoding this protein have been associated with schizophrenia and epilepsy, as well as brain malformations.
The scientists turned off MYT1L in human nerve cells grown from stem cells, leading to their hyperactivation observed in autism. This was because excess sodium channels were produced, which allowed sodium ions to enter the cell. Sodium is critical for electrical conductivity in the nerve cell, and excess sodium can cause neuron hyperactivation. Scientists used the drug lamotrigine to block these channels.
The researchers bred genetically modified mice that lack the MYT1L protein. The animals suffered from brain abnormalities and showed several autism-specific behavioral changes. These symptoms were significantly reduced when mice were given lamotrigine.
Scientists hope that drug therapy in adulthood can improve the function of brain cells in autism. However, the results are still limited to studies in mice. Clinical trials in patients with autism spectrum disorders are in the early stages of planning.