Research uncovers regenerative therapy for spinal cord injury
A drug that promotes nerve growth in adult human brain cells could offer a new way to aid recovery after spinal cord injury.
The compound, thiorphan, increased neurite outgrowth — the extensions that connect nerve cells — in adult human brain cells and improved hand function by 50 per cent in rats with spinal injuries.
Researchers from the University of California San Diego School of Medicine identified the drug using bioinformatics, comparing gene patterns linked to neuronal growth and regeneration against a large database of compounds.
Dr Erna van Niekerk is assistant project scientist in the department of neurosciences at UC San Diego School of Medicine and lead author of the study.
The researcher said: “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.”
Spinal cord injury often causes lifelong paralysis because neurons — the nerve cells that make up the brain and spinal cord — cannot regenerate effectively after damage. Encouraging this regrowth offers a promising treatment approach.
The team found that under specific experimental conditions, some mouse neurons activate genes involved in regeneration.
They then used computational analysis to find drugs that could trigger similar genetic activity.
Thiorphan, previously tested in humans for non-neurological conditions, emerged as a leading candidate.
Testing it in adult human brain cells marked a significant step forward, as these cells are notoriously difficult to culture in the lab.
In animal studies, rats treated with thiorphan alone showed a 50 per cent improvement in hand function after spinal injury compared with untreated animals. When combined with neural stem cell implants, recovery improved by another 50 per cent.
Van Niekerk described the discovery as “a convergence of technologies”.
She said: “Gene sequencing, computational bioinformatics and cell culture all came together to rapidly identify a potentially useful treatment that might have taken decades before these convergent technologies were available.”
Because thiorphan has already undergone safety testing in humans, the researchers say it could move quickly towards clinical trials for spinal cord injury.
They are now considering combining thiorphan with stem cell technology in upcoming trials.
“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,” said Dr Mark H. Tuszynski, professor in the department of neurosciences at UC San Diego and senior author.
“These are not stem cells; they are adult brain cells that previously were not possible to culture. The ability to culture adult brain cells could be useful for testing new drugs or gene therapies for many brain diseases.
“We are making efforts now to optimise thiorphan for future clinical trials, a task simplified by the fact that the drug has already been used safely in people,” van Niekerk added.
