Riluzole Cuts Spinal Cord Injury Damage by 30%

A drug called riluzole, commonly used to treat amyotrophic lateral sclerosis (ALS) and other motor neuron diseases, could also assist in recovery from spinal cord injuries, according to research from the University of Toronto's Temerty Faculty of Medicine.

The new study, published in eBioMedicine, showed that riluzole may offer benefits if taken within 12 hours of the injury occurring.

"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 Michael Fehlings, senior scientist at the Krembil Research Institute, University Health Network and professor of neurosurgery in the department of surgery at Temerty Medicine, where he is also vice-chair of research.

The findings are the result of a Ph.D. thesis by study co-author Karlo Pedro, now an assistant professor of surgery at Temerty Medicine and clinical associate at Toronto Western Hospital.

One of the most important takeaways from the research was the use of the Global Statistical Test (GST)—a new method of analyzing clinical trial results that enabled the data to be used in a way that tracked patients' outcomes at multiple stages, rather than a single end point.

The researchers say the test did a more comprehensive job of measuring the drug's total effect on patients by factoring in how riluzole affected things such as patients' neurological improvement, independent functioning and quality of life.

"I think this is a very helpful statistical technique," says Pedro. "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 said his work was based on data from the 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, and whom researchers followed for one year after the injuries happened. More than 80 percent of the patients were male.

Sixty five of the patients received riluzole about 12 hours after their injury for a 14-day period. The other 66 patients received a placebo.

When researchers measured how patients were doing six months after injury, they found that taking riluzole led to 58 percent "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 co-director of the spinal program at Temerty Medicine and holds 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."

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 assessing patient outcomes.

He offered 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.