One of the reasons SCI results in permanent disability is that the neurons that form our brain and spinal cord cannot effectively regenerate. Encouraging neurons to regenerate with drugs offers a promising possibility for treating these severe injuries.

The researchers found that under specific experimental conditions, some mouse neurons activate a specific pattern of genes related to neuronal growth and regeneration. To translate this fundamental discovery into a treatment, the researchers used data-driven bioinformatics approaches to compare their pattern to a vast database of compounds, looking for drugs that could activate these same genes and trigger neurons to regenerate.

Their approach identified Thiorphan—a drug previously tested in humans for non-neurological conditions—as a top candidate. The researchers successfully tested Thiorphan in adult human brain cells, finding that it increased neurite outgrowth, a key metric of regeneration. Being able to confirm that the drug works in adult human brain cells is a significant technical achievement, as brain cells are notoriously difficult to culture in the lab, making them virtually impossible to study in culture dishes.