Human trial of embryonic stem cell-based investigational drug for spinal cord injury begins

The first patient to be treated in an embryonic stem cell phase 1 clinical trial was enrolled at the Shepherd Center in Atlanta and received an investigational drug to treat an acute spinal cord injury, according to a press release from Geron Corporation, which is conducting the FDA-approved study.

The primary objective of the study is to assess the safety and tolerability of Geron’s human embryonic stem cell-derived oligodendrocyte progenitor cells, GRNOPC1, in patients with a complete American Spinal Injury Association Impairment Scale grade A subacute thoracic spinal cord injury (SCI). The injection of GRNOPC1, which contains low doses of human embryonic stem cells, must be done within 14 days of injury, according to Geron press releases.

In addition to the Shepherd Center, Northwestern Medicine in Chicago, is open for patient enrollment. Five other U.S. sites may be eligible to enroll patients in the clinical trial.

Milestone advancement

“Initiating the GRNOPC1 clinical trial is a milestone for the field of human embryonic stem cell-based therapies,” Geron president and CEO Thomas B. Okarma, MD, PhD, stated in the release. “This accomplishment results from extensive research and development of a succession of inventive steps to enable production of cGMP master cell banks, scalable manufacture of differentiated cell product, and preclinical studies in vitro and animal models of spinal cord injury, leading to concurrence by the FDA to initiate the clinical trial.”

Donald Peck Leslie, MD, medical director of Shepherd Center, said that his center was pleased to be involved in the research study. “We look forward to participating in clinical trials that may help people with SCI,” he stated in the release.

David A. Apple, MD, Shepherd Center medical director emeritus and former Orthopedics Today Editorial Board member highlighted the importance of the trial. “This clinical trial represents another step forward in Shepherd Center’s involvement in an attempt to find a cure for paralysis in people with SCI. Shepherd Center is an ideal place to conduct this study because of our clinical expertise and the volume of patients referred here for rehabilitation care,” he stated in the release.

Progress amidst controversy

The use of embryonic stem cells have been controversial since they are obtained from human embryos, but experts in the SCI field hold out hope that the cells may help regain some function in newly paralyzed patients.

In July, Geron was cleared by the FDA to proceed with its trial involving injection of GRNOPC1 directly into lesion sites in injured spinal cords. The FDA put the trial on hold following results of an animal study where Geron scientists observed a higher frequency of small cysts within the treated site than were previously reported, according to a company release.

Reference:
www.geron.com/GRNOPC1Trial

The technology that is being used for the Geron trial was invented by a research team led by Hans Keirstead, one of the scientists at the Reeve-Irvine SCI Center at University of California, Irvine. The trial is a safety study only. At best, patients and clinicians should only expect an incremental gain of function, but again, this trial will not be testing for efficacy. That being said, even an incremental gain of function means a great deal to an SCI patient.

The key aspect of this trial is that it is precedent setting for the stem cell field, and as such, will hasten the development of subsequent stem cell-based clinical trials.

– Ranjan Gupta, MD
Chair, Department of Orthopaedic Surgery
Professor of Orthopaedic Surgery, Anatomy & Neurobiology, and Biomedical Engineering
University of California, Irvine, Calif.

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