Researchers Fast-Track Spinal Cord Injury Drug Discovery Using Bioinformatics

Spinal cord injury (SCI) poses a significant medical challenge, frequently leading to lifelong paralysis and disability. Researchers at the University of California San Diego School of Medicine have made a breakthrough in addressing this issue by utilizing bioinformatics to expedite the discovery of a new drug aimed at treating SCI. This innovative approach not only holds promise for immediate treatment options but also streamlines the process for researchers worldwide to translate their findings into practical therapies.

One of the primary reasons for the permanence of disability following SCI is the inability of neurons in the brain and spinal cord to regenerate effectively. The UC San Diego team explored the potential of using drugs to stimulate neuronal regeneration, which could significantly improve outcomes for individuals with such injuries. Their research identified that, under specific experimental conditions, certain mouse neurons exhibited a unique gene expression pattern associated with growth and regeneration.

To leverage this discovery, the researchers employed data-driven bioinformatics techniques to analyze a comprehensive database of compounds. They sought drugs capable of activating the same regenerative genes. This analysis led them to identify Thiorphan, a drug previously tested in humans for non-neurological conditions, as a promising candidate for further exploration.

In laboratory tests, Thiorphan demonstrated the ability to enhance neurite outgrowth in adult human brain cells, marking a major technical achievement given the complexity of culturing these cells. The challenge of studying adult brain cells in laboratory conditions has historically hindered research in neurological disorders.

The team further evaluated Thiorphan’s effectiveness in animal models, specifically in rats with SCI. When combined with neural stem cell grafts, Thiorphan resulted in notable improvements in hand function and increased neuronal regeneration at the injury site. Rats treated solely with Thiorphan exhibited a remarkable 50% increase in recovery of hand function compared to untreated counterparts, with an additional 50% improvement when combined with the stem cell implant.

Looking forward, the researchers are considering the next steps for Thiorphan, with plans for clinical trials that will combine the drug with stem cell technology. Erna van Niekerk, Ph.D., assistant project scientist and lead author of the study, expressed enthusiasm for the findings, stating, “We were very happy to see that a drug that worked on cells in culture also showed effectiveness in an actual animal model of spinal cord injury. This is not always the case in new drug development.”

Van Niekerk noted that the identification of Thiorphan was the result of a “convergence of technologies,” including gene sequencing, computational bioinformatics, and cell culture techniques. This synergy allowed the team to rapidly identify a treatment that would have taken considerably longer without such advanced methodologies.

Another significant aspect of this research is that Thiorphan has already undergone safety testing in humans, facilitating a quicker transition into clinical trials for SCI. Mark H. Tuszynski, M.D., a professor in the Department of Neurosciences at UC San Diego and senior author of the study, emphasized the importance of their work, stating, “We succeeded in culturing adult human brain cells in large numbers in this study, offering a powerful new tool for the discovery of treatments for neurological disorders.”

The research was published in the journal Nature in March 2025 and received funding from various organizations, including the Adelson Medical Research Foundation, the Veterans Administration, the National Institutes of Health, and Wings for Life. The findings highlight the potential for technology to revitalize drug discovery processes by repurposing existing medications for new therapeutic applications, ultimately providing hope for improved treatments for spinal cord injuries.