New research shows drug holds promise for spinal cord injuries

University of Toronto researchers who treat people with spinal cord injuries have found that taking a drug called riluzole can help recovery, a new study says.  

The recent study in eBioMedicine used a new way to analyze clinical trial results, and, as a result, uncovered what researchers say is a “major advance” in treating severe spinal cord injuries. 

The research by Michael Fehlings and Karlo Pedro of the Temerty Faculty of Medicine showed taking riluzole — which is also used to treat Amyotrophic Lateral Sclerosis and other motor neuron diseases — may assist patients, if taken within 12 hours of the injuries happening. 

“This is a very valid and safe treatment option for clinicians and patients to consider. There’s quite rigorous control data now to show that,” says Fehlings, co-director of the spinal program and a professor in the division of neurosurgery at Temerty Medicine, as well as vice chair of research in the department of surgery. 

The findings in the study were the result of Pedro’s PhD thesis, which he did under Fehlings’ supervision.  

One of the most important takeaways of the research is the importance of using the Global Statistical Test (GST) to analyze clinical trial data, say Pedro and Fehlings.  

Researchers say that by using the GST, they were able to use the data in a way that tracked patients’ outcomes at multiple stages, rather than at a single end point of time.  

They also say the test did a more comprehensive job of measuring the drug’s total effect on patients, by factoring in how riluzole affected things like patients’ neurological improvement, independent functioning and quality of life.  

“I think this is a very helpful statistical technique,” says Pedro, an assistant professor at the department of surgery’s division of neurosurgery and a clinical associate at University Health Network’s Toronto Western Hospital.

“For the first time, we are able to show, supported by evidence, that a pharmacologic agent with a neuroprotective effect is able to treat severe spinal cord injuries. These findings will assist not only patients and clinicians, but researchers involved in future clinical trials.” 

Pedro moved to Toronto to work with Fehlings, and said his work was based on data from the original RISCIS (Safety and Efficacy of Riluzole in Acute Spinal Cord Injury Study) trial of 2023. 

The findings are based on data collected from 131 patients who had spinal cord injuries, who researchers followed for one year after the injuries happened. More than 80 per cent of the patients were male. 

Of the 131 total patients involved in the study, 65 received riluzole about 12 hours after their injury, for a 14-day period. The other 66 patients received a placebo, instead.

When researchers measured how patients were doing six months after injury, they found that taking riluzole led to 58 per cent “overall better outcomes” for people who took the drug, compared with the group who took the placebo.  

“An important takeaway for clinicians and people with spinal cord injuries is that this work demonstrates that the use of riluzole within 12 hours of an acute spinal cord injury improves outcomes, and that these outcomes are meaningful,” says Fehlings, who is also a clinician-scientist at Toronto Western Hospital’s Krembil Brain Institute and the Campeau/Tator Chair in Brain and Spinal Cord Research. 

“Our study looked at a patients’ motor recovery, combined with measuring their functional spinal cord independence and global health outcome quality of life,” says Fehlings. The drug is also inexpensive and leads to low side effects for patients, he adds.  

Fehlings says the study findings will be crucial when it comes to designing future clinical trials, and how to assess patient outcomes.

Fehlings used the example of a person who might regain the use of a finger after a spinal cord injury, and can then operate a phone or computer, which can have a dramatic effect on their quality of life.

“One of the challenges in many neurological conditions is that it’s very difficult to have one outcome measure that accurately assesses the multidimensional nature of recovery,” says Fehlings.

“What we have established here is a huge credit to Pedro, our statistical collaborator Peng Huang from Johns Hopkins University, and the international, multi-institutional contributors to this study," says Fehlings. "These findings reinforce the concept of looking at the multi-dimensional nature of recovery, by using the Global Statistical Test. They also ground the findings within the World Health Organization International Classification of Functioning, Disability and Health Framework, which is integral.”