Neuroscience discovery and development are undergoing dramatic change. Late stage clinical failures have forced the field to reevaluate the tools and hypotheses which have been used to design and develop drugs for central nervous system (CNS) disorders for the past 25 years. A number of key challenges have been identified:
Complex CNS diseases, which are caused by the interplay between many genes and pathways, are unlikely to be effectively treated using drugs which target a single protein.
The battery of standardized laboratory rodent models of behavior is far from optimal in predicting human clinical outcomes.
Clinical trials of CNS drugs are hampered by the lack of objective measures of disease state and drug effects.
At Galenea, we believe that each of these significant challenges can be addressed by a focus on synaptic transmission — the means by which signals are transmitted from neuron to neuron and across dense networks in the brain. Dysfunctions in synaptic transmission are key elements in Alzheimer’s disease (AD), Parkinson’s disease, Huntington’s disease, epilepsy, bipolar depression, schizophrenia, autism spectrum disorders, and traumatic brain injury. And in neurodegenerative disorders such as AD, changes at the synapse appear very early in the disease.