Brigham and Women’s Hospital, USA: Migulstat as a therapy

Miglustat as a therapy for secondary-progressive multiple sclerosis

Principal Investigator: Francisco Quintana, Ph.D.
Institution: Brigham and Women’s Hospital
Country: USA
Amount Awarded: €75,000

Astrocytes are cells in the brain that may play a destructive role in MS since among other activities, they are known to create scar tissue that may interfere with tissue repair. In the search for leads to new approaches to treating progressive MS, this team proposes to use an experimental mouse model that resembles several features of secondary-progressive MS to investigate the potential of a drug called miglustat. This drug is approved to treat other diseases, and it has been found to inhibit a protein, called “B4GALT6,” which the team found to be involved in the activation of astrocytes. The investigator proposes to test whether miglustat can inhibit astrocytes to stop disease progression. The team will also test its impact on human and mouse nerve cells isolated in the laboratory.

What does this mean for people living with progressive MS?

This study may point to a new strategy for stopping a key contributor to MS progression. Since miglustat is already is FDA-approved for other disorders, this would expedite its application to MS if it indeed proves safe and effective.


Project Update

Status: Complete

To date, in an animal model of MS, the team has found that daily administration of miglustat suppressed the clinical course of disease progression and reduced the recruitment of inflammatory  monocytes to the Central Nervous System. The data suggest that miglustat administration halts chronic EAE progression, and represents a potential therapeutic approach for progressive MS.


Principal Investigator Francisco J. Quintana

FFrancisco J. Quintana, PhD, earned his Diploma in Biology from the University of Buenos Aires and his doctoral degree in Immunology from the Weizmann Institute of Science in Rehovot, Israel. He completed postdoctoral training with a focus on Neuroimmunology at Brigham and Women’s Hospital, Harvard Medical School. He now conducts research focusing on the regulation of the adaptive and the innate immune response and biomarkers in Multiple Sclerosis and other immune-mediated diseases. His work in the field of Immunology has earned Dr. Quintana recognition from The Weizmann Institute of Science, the National MS Society, Nature Biotechnology, the Harvard Catalyst and the Tecan Group Ltd.

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