Multiple Sclerosis: Four game-changing insights from ECTRIMS 2025

Researchers and clinicians from across the field of Multiple Sclerosis (MS) converged at the 41st Congress of ECTRIMS in Barcelona this September. With over 9000 participants, more than 1,800 post presentations and 150 oral sessions the buzz was real - from breakthrough remyelination data to precision medicine strategies reshaping clinical trials.

Leone Atkinson , Executive Medical Director and Therapeutic Head of Neuroscience & Ophthalmology and Antoniya Todorova , Medical Director at Fortrea, bring you their brief reflections and top four highlights that should be on every neuroscience research director's radar.

1. Remyelination moves from theory to reality

This eagerly anticipated topic was presented as late-breaking news.The CCMR-Two trial delivered what the MS field has been waiting for: solid evidence that remyelination therapies can work^1,2. Using a combination of metformin and clemastine, researchers demonstrated meaningful myelin repair in progressive MS patients—a population that's historically been difficult to treat. This isn't just about one trial. It signals that remyelination strategies, including stem cell therapies and agents targeting oligodendrocyte precursor cells, are finally mature enough for serious clinical development. For sponsors planning progressive MS studies, this opens a new therapeutic pathway worth exploring.

2. Cognition deserves equal billing with physical outcomes

Maria Pia Amato's opening lecture put cognitive impairment front and center, emphasizing early detection and digital assessment tools³. What's particularly relevant for trial design: cognitive endpoints are becoming more standardized and measurable, thanks to advances in digital tools and validated batteries. Recent research demonstrates that tablet-based cognitive assessments like the Adaptive Cognitive Evaluation (ACE) show strong correlations with standard measures like the Symbol Digit Modalities Test, with the added benefit of remote, self-guided administration⁴. For sponsors, this means cognitive assessments should no longer be relegated to exploratory endpoints. They're increasingly expected by regulators, payers, and—most importantly—patients who want to know if treatments will preserve their ability to work and think clearly.

3. Precision medicine is no longer a buzzword in MS

Across the sessions, posters and presentations, it was clear that precision medicine has moved beyond theoretical frameworks into practical application. Genetic profiling, biomarker integration, and AI-driven analytics are actively reshaping how we design treatment strategies—and by extension, how we should be designing trials. Importantly, serum neurofilament light chain (sNfL) is emerging as a reliable marker for treatment response and disease activity⁵. The implications for clinical research are significant: patient stratification may become more sophisticated, target engagement may be established earlier, and we can potentially identify responders sooner.

4. Bruton's tyrosine kinase (BTK) inhibitors are proving their long-term value

Multiple sessions showcased compelling long-term efficacy and safety data for Bruton's tyrosine kinase (BTK) inhibitors⁶, particularly in progressive MS patients with limited treatment options^7,8. What emerged wasn't just another mechanism of action—it was evidence of sustained benefit in a population where we've historically struggled to show meaningful effects. For those planning trials in progressive MS, BTK inhibitors represent both a competitive landscape challenge and an opportunity to design smarter comparator studies.

Why this matters for your next protocol

ECTRIMS 2025 reinforced three critical shifts: MS research is successfully targeting disease mechanisms previously considered biologically inaccessible, precision medicine is enabling smarter trial design, and patient-centered outcomes—especially cognition—are becoming non-negotiable. The congress also highlighted ongoing challenges in global access and equity, reminding us that breakthrough science must ultimately translate to breakthrough care across diverse populations.

The digital platform keeps all sessions accessible through January 2026, so the learning continues. But the message from Barcelona is clear: the MS landscape is evolving fast, and clinical development strategies need to keep pace.

Ready to turn these insights into your next successful neuroscience trial? Let's talk about how Fortrea can help you navigate this evolving landscape.

References

1. Diabetes drug and antihistamine could together repair multiple sclerosis damage, trial finds. https://www.cam.ac.uk/research/news/common-diabetes-drug-and-antihistamine-could-together-repair-multiple-sclerosis-damage-trial-finds . Last updated 26 September 2025. Last accessed 08 October 2025.
2. CCMR Two: A Phase IIa, Randomised, Double-blind, Placebo-controlled Trial of the Ability of the Combination of Metformin and Clemastine to Promote Remyelination in People With Relapsing-remitting Multiple Sclerosis Already on Disease-modifying Therapy. https://clinicaltrials.gov/study/NCT05131828. Last updated 24 April 2025. Last accessed 08 October 2025.
3. Opening lecture at ECTRIMS 2025: Cognitive Function in Multiple Sclerosis: Progress and Perspectives. https://ectrims.eu/insights/ectrims-lecture-the-cognitive-dimension-of-ms/ . Last updated 17 July 2025. Last accessed 08 October 2025.
4. Hsu W, Rowles W, Anguera JA, et al. Assessing Cognitive Function in Multiple Sclerosis With Digital Tools: Observational Study. J Med Internet Res. 2022;24(1):e34007. doi:10.2196/34007.
5. Benkert P, Einsiedler M, et al. Differential dynamics of sGFAP and sNfL modelled over 30 years of MS disease: association with progression independent of relapse activity. Presented at ECTRIMS 2025. Abstract Number: 1809/O021.
6. Airas L, Bermel RA, et al. A review of Bruton's tyrosine kinase inhibitors in multiple sclerosis. Ther Adv Neurol Disord. 2024;17:17562864241233041.
7. Roche presents new data for OCREVUS and fenebrutinib across broad patient populations at ECTRIMS 2025. https://www.roche.com/media/releases/med-cor-2025-09-24 . Last updated 24 September 2025. Last accessed 08 October 2025.
8. Media Update: ECTRIMS: Sanofi showcases patient focus across multiple sclerosis. https://www.sanofi.com/en/media-room/press-releases/2025/2025-09-10-06-00-00-3147483 . Last updated 10 September 2025. Last accessed 08 October 2025.