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Asterias Biotherapeutics Reports Results from First-in-Man Clinical Trial of a Cell Therapy Derived from Embryonic Stem Cells

May 28, 2014

Asterias Biotherapeutics on May 22 presented the results of its Phase 1 clinical trial assessing the safety of its product, AST- OPC1, in subjects with spinal cord injury.

The results were presented by Dr. Jane Lebkowski, President of R&D of Asterias, at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in Washington, DC.

According to a company release, the study represented the first- in-man trial of a cell therapy derived from human embryonic stem cells (hESCs). The study was first initiated by Geron Corp. in 2010; in 2013 Asterias acquired all of Geron's stem cell assets. The results provide support for the safety of the product and provide a path for advanced clinical studies of AST-OPC1. The full contents of Dr. Lebkowski's presentation are available on Asterias' website.

The company reported that AST-OPC1 is a population of cells derived from hESCs that contain oligodendrocyte progenitor cells (OPCs). OPCs and oligodendrocytes provide several important supportive functions for nerve cells in the central nervous system. In the phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury as classified by the American Spinal Association Impairment Scale, were administered a relatively low dose of two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. The subjects received low levels immunosuppression for the next 60 days. The patients have been followed to date for 2-3 years through numerous clinical visits, MRIs, and neurological assessments. The trial was conducted by Dr. David Apple at the Shepherd Center in Atlanta GA, Dr. Richard Fessler at Northwestern University in Chicago IL, and Drs. Gary Steinberg and Stephen McKenna at Stanford University/Santa Clara Valley Medical Center in Palo Alto, CA.

Additionally, delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with the intraoperative administration of the cells. In addition, there were no serious adverse events associated with AST-OPC1 itself, or the immunosuppressive regimen. There was no evidence of expanding masses, expanding cysts, infections, cerebrospinal fluid leaks, increased inflammation, or neural tissue deterioration at the injury site of these subjects. Immune monitoring of subjects through one year post-transplantation showed no evidence of antibody-based or cellular immune responses to AST-OPC1, despite complete withdrawal of all immunosuppression at sixty days post-transplant. In four of the five subjects, serial MRI scans performed throughout the 2-3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration. This effect was seen in the animal model testing of AST-OPC1. There were no unexpected neurological degenerations or improvements in the five subjects in the trial as evaluated by the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) exam. Patients in the trial will be followed for a total of 15 years.

There have been three serious adverse events observed in the trial that were not associated with AST-OPC1, its delivery or the transient immunosuppression, but which are common in spinal cord injury patients. These included one each of pyelonephritis, urinary tract infection, and dyspnea due to autonomic dysreflexia. There were no deaths in the trial.

"We are grateful to our patients, clinical investigators and their institutions for their tremendous support throughout the execution of this first-in-human trial of a human embryonic stem cell based therapy," said Dr. Edward Wirth III, Chief Translational Officer of Asterias.

"The safety demonstrated in this trial positions Asterias to start a new phase 1/2a clinical trial in 2014, subject to clearance from the FDA," said Dr. Lebkowski.

"Spinal cord injury represents a tremendous unmet medical need that not only results in severe disability, but can also significantly shorten the projected lifespan of affected individuals," said Dr. Stephen McKenna, Director of the Rehabilitation Trauma Center, Santa Clara Valley Medical Center. "There are no approved therapies that can repair spinal cord injuries. We look forward to participating in the future clinical development of AST-OPC1."

Asterias Biotherapeutics is a biotechnology company focused on the field of regenerative medicine.

BioTime is a biotechnology company engaged in research and product development in the field of regenerative medicine.

Shepherd Center is a private, not-for-profit hospital specializing in medical treatment, research and rehabilitation for people with spinal cord injury or brain injury.

Santa Clara Valley Medical Center is a public hospital located in Silicon Valley.

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