AST-OPC1 Stem Cell Therapy May Offer New Hope for Spinal Cord Injury

Hand function returning in some patients

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An investigational treatment called AST-OPC1 (oligodendrocyte progenitor cells) may give new hope to people with a recent spinal cord injury. Researchers are examining whether AST-OPCI injected directly into the spinal cord helps repair damage in people with cervical (neck) spinal cord injury.

brain and upper central nervous system, spinal cordResearchers are examining whether AST-OPCI injected directly into the spinal cord helps repair damage in people with cervical (neck) spinal cord injury. Photo Source:“Until now, there have been no new treatment options for the 17,000 new spinal cord injuries that happen each year,” said primary investigator Richard G. Fessler, MD, PhD, Professor of Neurological Surgery at Rush University Medical Center, Chicago, Illinois. “We may be on the verge of making a major breakthrough after decades of attempts.”

AST-OPC1 is developed from stem cells and is believed to work by supporting the proper functioning of nerve cells. After a spinal cord injury, many nerve cells are severed and beyond repair; however, many nerve cells have the potential to work again but have lost their protective coating (known as myelin) that helps nerves transfer messages to the arms and legs.

“What AST-OPC1 does is recoat those potentially functional cells and allows them to work more normally,” Dr. Fessler told SpineUniverse.

normal myelin sheath and damaged Left: Normal myelin sheath Right: Damaged myelin sheath. Photo Source:

Three Doses of AST-OPC1 Are Being Studied

Dr. Fessler and colleagues are part of a larger multicenter trial designed to assess the safety and effectiveness of three doses of AST-OPC1 (2-, 10-, or 20-million cells) injected into the injured area of the spinal cord between 14 and 30 days following a cervical spinal cord injury. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the arms and legs.

Thus far, Dr. Fessler and colleagues have injected three patients at the first dose level and five patients at the intermediate dose level.

Hand Function Is Returning in Some Patients

“Our preliminary results show that we may, in fact, be getting some regeneration. Some of those who have lost use of their hands are starting to get function back. That is the first time in history that has ever been done,” Dr. Fessler said. The improvements are seen within the 30 to 60 days, he noted.

“I have been doing this kind of research for more than 20 years, and I’ve never seen anything as encouraging as AST-OPC1,” Dr. Fessler said. “Just as a journey of a thousand miles is done one step at a time, repairing spinal cord injuries is being done one step at a time. And, now, we can say that we’ve taken that first step.”

The injections are safe, as determined by an earlier study of AST-OPC1 that involved patients with thoracic (mid-back) spinal cord injury. Dr. Fessler said it important for the spinal cord injury to be recent in order for the therapy to work. In addition, the spinal cord needs to be in continuity and not severed. The injections are unlikely to be effective in people who have had spinal cord injuries for years, although future trials are needed to know for sure.

Updated on: 08/01/19
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Spinal Cord Injury: Nerve Regeneration and Stem Cells
Richard G. Fessler, MD, PhD
Professor of Neurosurgery
Rush University Medical School
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