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Paralysis from Rare Disorder: Stem Cell Therapy Trial Brings Hope

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By injecting patients with stem cells engineered to repair the central nervous system – called progenitor cells – scientists are working to establish the first treatment that can repair spinal cords inflamed by transverse myelitis.

If successful, the clinical trial could lead to similar therapies for more common conditions such as multiple sclerosis.

“The trial has been 15 years in the making with a huge number of hurdles,” said Dr. Benjamin Greenberg, explaining the challenges of developing cells that could both find the damaged area and fix the problem.

Paralyzing disease

Transverse myelitis is caused by an inflammation in the spinal cord that damages myelin, a protective coating around neurons. The damage inhibits communication between nerve fibers in the spinal cord and the rest of the body, resulting in partial or total paralysis. Most patients at least moderately recover within a few months, while a slim minority face permanent paralysis.

UT Southwestern’s clinical trial will study the safety and effectiveness of implanting these cells into the spinal cord. Ultimately, the hope is to reverse paralysis.

The trial will begin with nine participants with the most severe form of transverse myelitis. Each will receive a one-time injection of progenitor cells designed to produce myelin along the damaged area and re-establish critical nerve signaling.

The cells have successfully repaired the central nervous system in animals and could establish new treatments for other disorders involving damaged myelin. This includes multiple sclerosis and an array of brain and spinal cord conditions such as neuromyelitis optica.

About the clinical trial

Q Therapeutics, a developer of clinical-stage cell therapies for central nervous system diseases, is providing the progenitor cells and trial support. The Transverse Myelitis Association, a nonprofit advocacy organization dedicated to rare neuro-immune disorders, will help locate participants and facilitate their travel to Dallas. The trial is funded in part by Q Therapeutics, the Transverse Myelitis Association, the Lyda Hill Foundation, and the M.R. and Evelyn Hudson Foundation.

The trial is overseen by Dr. Greenberg, who leads the Conquer programs at UT Southwestern, including the Transverse Myelitis and Neuromyelitis Optica Program and the Pediatric Conquer Program at Children’s Medical Center Dallas. He is an Associate Professor of Neurology & Neurotherapeutics and Pediatrics, a Distinguished Teaching Professor, and a Cain Denius Scholar in Mobility Disorders.

This article has been republished from materials provided by the University of Texas Southwestern Medical Center. Note: material may have been edited for length and content. For further information, please contact the cited source.