Accelerating Therapeutic Development in Pediatric Multiple Sclerosis: Brenda Banwell, MD

WATCH TIME: 5 minutes

"For every 25 therapies approved for adults, we have just one or two for pediatric-onset MS. We must design smarter, faster, and more feasible trials to close that gap and bring therapies to children sooner."

Pediatric multiple sclerosis (MS) is a chronic, immune-mediated demyelinating disease of the central nervous system that begins before 18 years of age, accounting for approximately 3%-5% of all MS cases. To date, there are only a small handful of approved therapies specific to treat children and adolescents with the disease. Ocrelizumab (Ocrevus; Genentech), the only FDA-approved therapy for progressive MS, is currently being evaluated as a potential treatment for pediatric, relapsing patients through the phase 3 OPERETTA 2 trial (NCT05123703).

The trial includes 187 patients with pediatric MS, aged 10-17 years, who are randomized to either ocrelizumab 600 mg every 24 weeks or daily oral 0.5 mg fingolimod (Gilenya; Novartis), with matching placebos, over a double-blind period lasting 24 weeks. New data from the study presented at the 2025 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, held September 24-26, in Barcelona, Spain, revealed that ocrelizumab met its primary end point of noninferiority to fingolimod in annualized relapse rate reduction. In addition, ocrelizumab outperformed fingolimod in suppressing new or enlarging T2 and gadolinium-enhancing lesions.

The data, presented by Brenda Banwell, MD, marked a step forward in the care for children with MS. Following the meeting, Banwell, pediatrician-in-chief at Johns Hopkins Children’s Center, discussed the challenges and future directions for advancing research in pediatric MS. In the discussion, she highlighted how pediatric trials lag 5 to 10 years behind adult approvals, leaving limited options available for this small yet vulnerable population. Furthermore, she spoke on key barriers such as low disease prevalence, family burden, and competition with adult studies, while advocating for earlier inclusion of adolescents in phase 3 trials and closer collaboration among researchers, regulators, and pharmaceutical sponsors.

Click here for more ECTRIMS 2025 coverage.

REFERENCE
1. Banwell B, Kotulska-Jozwiak K, Rostásy K, et al. Efficacy and safety of ocrelizumab compared with fingolimod in paediatric relapsing-remitting MS: results of the Phase III OPERETTA 2 study. Presented at ECTRIMS Congress; September 24-26, 2025; Barcelona, Spain. Late-Breaking Abstract O130.