AST-OPC1 is comprised of oligodendrocyte progenitor cells manufactured from our pluripotent embryonic stem cell platform.
The AST-OPC1 stem cell lines were derived from an embryo that was originally meant for an in vitro fertilization procedure in the late 1990's. The embryo was no longer needed by the couple, however, and the embryo was going to be discarded. The couple consented to donate that embryo and the cell line was derived from it. Of note, this cell line was formally approved as "ethically derived" by both the Bush and Obama administrations. Importantly, Asterias has never used fetal tissue of any kind in any of its research programs. Asterias has banks of these embryonic stem cells, derived from this cell line, which the company can multiply to make cells as needed and, in turn, convert them into oligodendrocyte progenitor cells for use in clinical trial evaluation in patients. The cell line used by Asterias, which was originally derived in the 1990s, is sufficient to supply ALL of the AST-OPC1 needed for clinical trials and for potential commercialization of the product following regulatory approval.
The clinical program is testing the utility of AST-OPC1 in spinal cord injury patients. A large body of preclinical studies has demonstrated the safety and efficacy of AST-OPC1 in models of thoracic (back) and cervical (neck) spinal cord injury. These potential indications are currently being evaluated through preclinical studies.
Four important reparative functions of AST-OPC1 were observed in these studies:
Restoration of the protective "myelin" coating on nerve cells
Production of factors that stimulate nerve cell growth
Recruitment of blood vessels to
deliver oxygen & nutrients
to the site
Reduction of the size of the injury cavity
Importantly, significant improvements in locomotor (walking) function were seen with AST-OPC1 transplantation in models of both thoracic and cervical injuries. These improvements included increases in running speed, right forelimb stride length, right forelimb maximal longitudinal deviation, and right rear stride frequency.
We are currently enrolling patients in a phase 1/2a open-label, single-arm study (the SCi-Star study) testing three escalating doses of AST-OPC1, starting with 2 million cells, escalating to 10 million and then 20 million cells. The protocol criteria includes patients with sub-acute cervical spinal cord injury (SCI), last fully preserved neurological level of injury from C5-C7, ages 18-69. Investigators will evaluate the safety of AST-OPC1 administered once between 14 and 30 days after injury and the impact on hand and arm function.