Spinal cord injury (SCI) is a devastating event resulting in permanent loss of neurological function. To date, effective
therapies for SCI have not been established. With recent progress in neurobiology, however, there is hope that
drug administration could improve outcomes after SCI.
Riluzole is a
benzothiazole anticonvulsant with
neuroprotective effects. It has been approved by the U.S. Food and Drug Administration as a safe and well-tolerated treatment for patients with
amyotrophic lateral sclerosis. The mechanism of action of
riluzole involves the inhibition of pathologic glutamatergic transmission in synapses of neurons via
sodium channel blockade. There is convincing evidence that
riluzole diminishes neurological tissue destruction and promotes functional recovery in animal SCI models. Based on these results, a phase I/IIa clinical trial with
riluzole was conducted for patients with SCI between 2010 and 2011. This trial demonstrated significant improvement in neurological outcomes and showed it to be a safe
drug with no serious adverse effects. Currently, an international, multi-center clinical trial (
Riluzole in Acute
Spinal Cord Injury Study: RISCIS) in phase II/III is in progress with
riluzole for patients with SCI (clinicaltrials.gov, registration number NCT01597518). This article reviews the pharmacology and neuroprotective mechanisms of
riluzole, and focuses on existing preclinical evidence, and emerging clinical data in the treatment of SCI.