The Foundation’s External Awards Program has selected a new project for funding in the final quarter of 2018. Alan Verkman, MD, PhD, Professor of Medicine and Physiology in the Department of Medicine at the University of California San Francisco, will conduct a study entitled, “Focused ultrasound for therapy of neuromyelitis optica – an inflammatory demyelinating disease of the central nervous system.”
Dr. Verkman and his research team seek to determine whether they can deliver antibody and cell therapeutics across the blood-brain barrier to reduce neuroinflammation and promote remyelination in this disease.
This project will advance antibody and cell therapeutics for neuromyelitis optica, an autoimmune inflammatory disease of the central nervous system associated with demyelination and neurological impairment. The disease is caused by autoantibodies that bind to astrocyte protein aquaporin-4. Focused ultrasound will be used to transiently open the blood-brain barrier to deliver antibody and cell therapeutics, including a therapeutic antibody (aquaporumab) that blocks autoantibody binding to aquaporin-4, and stem cells that promote remyelination. The methods established in this project may be widely applicable to delivery of large molecule and cell therapeutics into the central nervous system for treatment of neurological diseases