While dramatic improvements have been made in our ability to diagnose neurodegenerative diseases, effective treatment options are lagging behind. Even targeted genetic strategies for monogenic disorders have struggled to be effective without toxicity. Advancing therapeutics will require a shift in the conventional platforms used for drug discovery, specifically exploiting those that incorporate more elements of human disease. This may include reframing the focus of neurodegeneration beyond the neuron. Platforms beyond simple neuronal cultures, including glial cell integration, a three-dimensional architecture, a blood-brain barrier, and recapitulation of dysfunctional circuits, are more likely to lead to therapeutic success.

Some techniques lend themselves easily for high-throughput screening against chemical libraries, while others are better served to validate lead molecules. Examples of methods to include in this collection are automated microscopy, patient-derived stem cells and CNS organoids, isolation and examination of oligodendrocytes or other glial cells, high-throughput screening against ion channels, novel delivery mechanisms for small molecules and gene-based therapies, and behavioral phenotyping in vertebrate models.