Translating Stem Cell Models Into Clinical Insights in Multiple Sclerosis: Valentina Fossati, PhD

WATCH TIME: 4 minutes

“We really need to integrate stem cell models with detailed clinical data and long-term patient follow-up. Other fields like Alzheimer and Parkinson disease are already doing this, and I think the MS community should be open to learning from those approaches.”

Efforts to better model multiple sclerosis (MS) in the laboratory have expanded significantly in recent years, particularly as researchers seek systems that more accurately reflect human central nervous system biology. While experimental autoimmune encephalomyelitis and postmortem tissue studies have provided foundational mechanistic insights, both approaches carry limitations when attempting to dissect cell-specific pathology or capture patient-level heterogeneity.¹ As a result, induced pluripotent stem cell (iPSC) platforms have emerged as a complementary strategy, enabling investigators to generate patient-derived neural and glial cells for mechanistic interrogation in controlled systems.²

At theAmericas Committee for Treatment & Research in Multiple Sclerosis (ACTRIMS) Forum 2026, held February 5-7, 2026, in San Diego, California, Valentina Fossati, PhD, senior research investigator at the New York Stem Cell Foundation, presented on how iPSC technology is reshaping disease modeling in MS. Her work focuses on producing oligodendrocytes, astrocytes, microglia, and neurons from patient-specific lines and incorporating them into co-culture systems and 3-dimensional organoids designed to better approximate human brain architecture. These platforms allow for the study of glial crosstalk, inflammatory signaling, and myelination dynamics in ways that extend beyond traditional models.³

During the meeting, Fossati sat down with NeurologyLive^® to discuss what additional research efforts are needed to advance these models toward translational impact. In the conversation, she outlined the importance of integrating stem cell platforms with longitudinal clinical cohorts, deep phenotyping, and lessons from Alzheimer and Parkinson disease research. She also addressed the remaining technical limitations, including incomplete vascular and immune system modeling, and how continued refinement may help bridge experimental findings with clinical care.

Click here for more ACTRIMS Forum 2026 coverage.

REFERENCES
1. Robinson AP, Harp CT, Noronha A, Miller SD. The experimental autoimmune encephalomyelitis model of multiple sclerosis: utility for understanding disease pathophysiology and treatment. Handb Clin Neurol. 2014;122:173-189. doi:10.1016/B978-0-444-52001-2.00008-X
2. Dolmetsch R, Geschwind DH. The human brain in a dish: the promise of iPSC-derived neurons. Cell. 2011;145(6):831-834. doi:10.1016/j.cell.2011.05.034
3. Pamies D, Barreras P, Block K, et al. A human brain microphysiological system derived from induced pluripotent stem cells to study neurological diseases and toxicity. ALTEX. 2017;34(3):362-376. doi:10.14573/altex.1609122