All roads lead to Rome, but which one is the best? The direction of Autism Spectrum Disorder drug development.
Summary
Autism Spectrum Disorder (ASD) is a mental disorder that affects roughly 1% of the population. The main symptoms of ASD are poor communication, poor social interactions and abnormal behavior such as repetitive behavior.
Recently, research to drug development for ASD has received a great amount of interest, due to increasing knowledge about proteins and pathways involved in ASD. These pathways mainly include protein synthesis regulation systems, such as the mGluR5-pathway and the endocannabinoid system. Protein synthesis is highly important during brain development for cell migration and synaptogenesis, but also in the mature brain to maintain the balance between excitatory and inhibitory synapses and to make synaptic plasticity possible. These processes are all highly important for cell communication and thereby brain functioning.
The insight that processes in the mature brain also contribute to ASD provide the possibility to treat ASD at later ages, whereas the initial conception about ASD was that it is a developmental disorder that only could be treated at a very young age. This highly increased the interest of many researchers into ASD drug development.
Currently, there are clinical trials running with drugs that target the core pathways of ASD. Although the results are promising, one should take into account that many of these drugs also target peripheral cells and therefore may cause side effects on the long run. Therefore, the future of the direction of drug development remains uncertain. In addition, money issues, politics and sharing knowledge may be important issues to consider regarding to drug development.