VIDEO: Troriluzole alters trajectory of spinocerebellar ataxia

DENVER — Four studies presented at the American Academy of Neurology annual meeting show improvements in patients with spinocerebellar ataxia who take troriluzole.

“SCAs [spinocerebellar ataxias] are, at present, untreatable, inexorably progressive diseases,” Jeremy Schmahmann, MD, said in this Healio video. “We’re hoping for a therapeutic intervention that will modify the course of the disease over time.”

The four studies presented collectively show an altered trajectory in patients taking troriluzole, according to Schmahmann, founding director of the Massachusetts General Hospital Ataxia Center and director of the hospital’s Laboratory for Neuroanatomy and Cerebellar Neurobiology.

“Patients fall less, they seem to progress at a slightly slower pace, the video assessments seem to be effective, the f-SARA [modified-functional Scale for the Assessment and Rating of Ataxia] seems to have patient relevance, and the SCACOMS [Spinocerebellar Ataxia Composite Scales] seems to provide confidence that, in fact, these data are accurate,” he said.

References:

• Beiner M, et al. Automated video-based characterization of movement quality in a phase III clinical trial of troriluzole in subjects with spinocerebellar ataxia. Presented at: American Academy of Neurology annual meeting; April 13-18, 2024; Denver.
• Beiner M, et al. Matching-adjusted indirect comparison of troriluzole versus untreated natural history cohort in spinocerebellar ataxia. Presented at: American Academy of Neurology annual meeting; April 13-18, 2024; Denver.
• L'Italien G, et al. Development of a novel composite measure (SCACOMS) to assess disease progression in spinocerebellar ataxia. Presented at: American Academy of Neurology annual meeting; April 13-18, 2024; Denver.
• Potashman M, et al. Psychometric validation of the modified-functional scale for the assessment and rating of ataxia. Presented at: American Academy of Neurology annual meeting; April 13-18, 2024; Denver.