(WHAM) - A study based on 15 years of research at the University of Rochester Medical Center is providing progress in a potential treatment for multiple sclerosis patients.
The study published in the medical journal Cell Reports showed how specific human brain cells can repair damage and restore function when transplanted into mice that modeled multiple sclerosis and other white matter diseases.
The treatment, developed by a University of Rochester start-up company called Oscine Therapeutics, can now move forward to clinical trials.
“These findings have significant therapeutics implications and represent a proof-of-concept for future clinical trials for multiple sclerosis and potential other neurodegenerative diseases," said Dr. Steve Goldman, professor of Neurology and Neuroscience at the University of Rochester Medical Center.
The study focuses on support cells called glia that are found in the brain.
Dr. Goldman, who was the lead author on the study, oversaw the lab that developed techniques to manipulate the chemical signaling of embryonic and induced pluripotent stem cells to create glia.
In the autoimmune disorder multiple sclerosis, glial cells are lost as the disease progresses. As MS progresses, it causes the body's immune system to specifically attack special cells called oligodendrocytes that create a substance called myelin. Myelin plays an important role in helping nerve cells communicate with one another by serving as a sort of insulation for nerve fibers as impulses are sent across them.
As myelin is lost during the progression of MS, signals between nerve cells becomes disrupted.
The new study showed how transplanted human glia cells migrated where they were needed in the brain, which created new oligodendrocytes, and replaced the lost myelin.
The experimental transplant therapy is currently under early FDA review for clinical trials.
Martha Windrem, the study's first author, Steven Schanz, Lisa Zou, Devin Chandler-Militello, Nicholas Kuypers, Maiken Nedergaard, Yuan Lu, and John Mariani all worked at URMC on the study.
Funding 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 supported the research.