Immunosuppressant Prodrug Shows Potential as Acute Treatment for Individuals Experiencing Severe MS Relapses

Cyclophosphamide, an immunosuppressant prodrug, may provide meaningful benefit for people with multiple sclerosis (MS) who are experiencing severe relapses unresponsive to standard treatments such as corticosteroids or plasma exchange, according to new data from a clinic-based cohort study presented at the 41st Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS). In the study, cyclophosphamide was associated with improvements in disability scores, number of relapses within 1 year, and new MRI activity, with no serious adverse events reported.

Researchers reviewed data for 23 individuals from the University of British Columbia MS clinic who received cyclophosphamide treatment between 2009 and 2024 and met the definition for MS diagnosis according to 2017 McDonald criteria. Clinical response was assessed by changes in Expanded Disability Status Scale (EDSS) scores at baseline and 12 months after treatment, along with MRI evaluation for new T2 lesions. Safety outcomes were also monitored.

Key results included:

• A total of 13 participants showed improvements in in EDSS scores; 5 improved by ≥3 points.
• No participants experienced relapses reported within 1 year posttreatment.
• Two participants with highly active MS developed new T2 lesions within 1 year of completing treatment with cyclophoshamide; both of whom had demonstrated recovery after treatment with cyclophosphamide.
• In terms of adverse events, 1 participant had sinus infection, 1 had dental infection, and 1 had epididymitis; these 3 cases of infection were successfully treated using antibiotics.
• There were no reported serious adverse events.

The authors of this study are affiliated with the University of British Columbia MS MS/Neuromyelitis Optica (NMO) Clinic.