Summary: Transplanting human glial progenitor cells derived from induced pluripotent stem cells into animal models of multiple sclerosis promotes repair of damaged white matter and restores neurological function.
Source: University of Rochester
Overview: A new study from the University of Rochester reports that specific human brain cells transplanted into animal models of multiple sclerosis (MS) and other white matter disorders can replace lost support cells, rebuild myelin, and restore function. The findings provide key preclinical evidence supporting the advancement of this cell-based therapeutic approach toward human clinical trials.
Steve Goldman, M.D., Ph.D., professor of Neurology and Neuroscience at the University of Rochester Medical Center (URMC), co-director of the Center for Translational Neuromedicine, and the study’s lead author, said, “These results show that transplantation of human glial cells can achieve remyelination in the adult brain.” He emphasized that the work represents an important proof-of-concept with significant therapeutic implications for multiple sclerosis and potentially other neurodegenerative conditions.
The research, published in the journal Cell Reports, builds on more than 15 years of URMC research into glia—non-neuronal cells that support brain structure and function. Goldman’s laboratory has refined methods to direct both embryonic and induced pluripotent stem cells (iPSCs) to become glial progenitor cells. These progenitors differentiate into the brain’s principal support cells, including astrocytes and oligodendrocytes, which maintain neural health and enable rapid signal transmission.
In MS, the immune system attacks oligodendrocytes and the myelin they produce. Myelin is the insulating sheath that surrounds nerve fibers and facilitates efficient electrical signaling. When myelin is damaged or lost, neuronal communication breaks down, leading to the sensory, motor, and cognitive impairments that characterize MS. Early in the disease—during relapsing-remitting stages—some remyelination can occur as endogenous oligodendrocytes repair damaged sheaths. Over time, however, the brain’s capacity to regenerate myelin declines, and the disease progresses to irreversible disability.
In the current study, URMC researchers transplanted human glial progenitor cells into adult mice that model progressive MS. The transplanted cells migrated through white matter regions, matured into oligodendrocytes, and formed new myelin around denuded axons. Importantly, this remyelination correlated with measurable recovery of motor function in the treated animals. The results indicate that human glial cell transplantation can not only replace lost cells but also restore neural circuitry and improve behavior in a disease-relevant setting.
Beyond multiple sclerosis, the authors note that this cell-based approach could be relevant for other disorders of white matter. Potential applications include pediatric leukodystrophies—hereditary conditions in which myelin fails to develop properly—and certain types of stroke that damage adult white matter. The broad concept is to use patient-compatible iPSC-derived glial progenitors as a source for tissue repair in diverse demyelinating and glial diseases.
This line of research is being translated toward clinical use through a University of Rochester start-up, Oscine Therapeutics, which is developing experimental transplant therapies for MS and other glial disorders, including Huntington’s disease. Oscine’s early-stage cell therapy program is under preliminary review by the U.S. Food and Drug Administration for clinical trial consideration. Steve Goldman is the scientific founder of Oscine, serves as an officer, and holds equity in the company.
Co-authors on the paper include first author Martha Windrem, along with Steven Shanz, Lisa Zou, Devin Chandler-Militello, Nicholas Kuypers, Maiken Nedergaard, Yuan Lu, and John Mariani, all affiliated with URMC.
Funding: The study received support from the National Institute of Neurological Disorders and Stroke, the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation, the Mathers Charitable Foundation, the New York Stem Cell Research Program (NYSTEM), the Oscine Corporation, and Sana Biotechnology. Dr. Goldman also holds an appointment at the University of Copenhagen, where his work is supported by the Novo Nordisk Foundation and the Lundbeck Foundation.
About this neuroscience research article
Source:
University of Rochester
Media Contacts:
Mark Michaud – University of Rochester
Image Source:
The image is in the public domain.
Original Research: The study will appear in Cell Reports.
These findings strengthen the scientific rationale for advancing human glial progenitor cell transplantation as a targeted remyelination therapy. By combining patient-derived iPSCs, directed differentiation into glial lineages, and rigorous preclinical testing, this approach aims to restore myelin, protect neural networks, and ultimately improve outcomes for people affected by multiple sclerosis and other white matter disorders.
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