AST-OPC1 is an oligodendrocyte progenitor cell-based therapy being developed by our majority-owned subsidiary Asterias Biotherapeutics, Inc. (NYSE MKT: AST). AST-OPC1 is now in a Phase I/IIa clinical trial in patients with complete cervical spinal cord injury, and is funded in part through a $14.5 million grant from the California Institute for Regenerative Medicine (CIRM). The goal of this clinical trial is to test the safety of the product in the cervical region of the spine and to escalate the dose in human patients to levels comparable to those already demonstrated as effective in an animal model of the disorder. Asterias is currently conducting a Phase I/IIa human clinical trial of AST-OPC1 on patients with cervical spinal cord injury.
A Phase I trial has already been completed. In this trial, AST-OPC1 was administered to five patients with neurologically complete, thoracic spinal cord injury. The trial was led by principal investigator Richard G. Fessler, MD, PhD, professor of neurological surgery at Rush University Medical Center.
In the trial, the five patients were administered two million AST-OPC1 cells at the site of the spinal cord injury 7 to 14 days post-injury. The patients then received low levels of an immunosuppressant for the next 60 days.
The trial’s primary endpoints of safety and feasibility were met and follow-up studies have shown no serious side effects from the therapy.
“Importantly, all five subjects were followed for over five years after receiving the two million AST-OPC1 cells. In four of the five subjects, MRI scans have shown that the actual injury site had shrunk and that the AST-OPC1 cells may have had some positive effects in reducing the deterioration of spinal cord tissue.” – Richard G. Fessler, M.D., Ph.D.
Ongoing Phase I/IIa Trial for Spinal Cord Injury
The first injury to be targeted for AST-OPC1 is complete cervical spinal cord injury (SCI). In March 2015, Asterias initiated patient enrollment in a new Phase I/IIa trial with Shepherd Center. Based in Atlanta, Shepherd Center is one of the nation’s top rehabilitation hospitals for spinal cord and brain injury. The trial is being led by principal investigator Donald Peck Leslie, M.D., the medical director of Shepherd Center. The Phase 1/2a clinical trial is designed to assess safety and activity of escalating doses of AST-OPC1 for complete cervical SCI. The trial is an open-label, single-arm study testing three escalating doses of AST-OPC1 in patients with sub-acute, C-5 to C-7, neurologically complete cervical SCI.
These patients have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 is being administered to them 14 to 30 days after injury. After treatment, patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product.
In the first half of 2015, Asterias announced an initiative to accelerate the timelines for the trial by approximately six months. In addition, the company plans to seek FDA concurrence to increase the robustness of the proof of concept in the trial by expanding enrollment from 13 patients to up to 40 patients.
Asterias also plans to enroll patients for the trial in up to seven additional sites in coming months.
Additional information about the trial may be found at www.asteriasbiotherapeutics.com and at www.clinicaltrials.gov.
Scientific references for AST-OPC1 may be found here.
Significant Unmet Need with No Approved Therapies or Devices
- More than 12,000 people sustain a spinal cord injury every year in the United States
- 1.3 million Americans are estimated to be living with a spinal cord injury
- Cost of care of an individual with spinal cord injury averages in the range of $3 million to $4 million over a lifetime, in addition to the loss of income
Asterias Biotherapeutics’ products and technologies are protected by a strong portfolio of intellectual property developed internally and licensed from other entities.
Click on the files below to view Asterias’ patents and trademarks, for AST-OPC1 and other programs and technologies.
BioTime Company Information
Asterias Biotherapeutics is in the BioTime family of companies and is traded on the New York Stock Exchange Market Exchange, ticker symbol (NYSE MKT: AST).
Asterias is led by chief executive officer Pedro Lichtinger, president and chief executive officer; Jane S. Lebkowski, Ph.D., president of research and development; Katherine Spink, Ph.D., chief operating officer; and Edward D. Wirth, III, M.D., Ph.D., chief translational officer.
Asterias has significant support from the California Institute of Regenerative Medicine (CIRM). In May 2014 CIRM granted Asterias a $14.3 million Strategic Partnership Award to re-initiate this trial, the world’s first embryonic stem cell-based human clinical trial, for spinal cord injury. Supported by California public funds, CIRM is the largest funder of stem cell-related research and development in the world.
More information can be located at www.asteriasbiotherapeutics.com.