Pioneering Cell Transplants for the Treatment of Spinal Cord Injury

Contributed by Lineage Cell Therapeutics

Each year, spinal cord injury (SCI) affects an estimated 18,000 individuals in the United States, with less than 1% of those experiencing complete neurologic recovery upon hospital discharge. Current treatments primarily focus on rehabilitation, assistive devices, medications, and surgery; however, these only provide benefits in mitigating symptoms, improving mobility, stabilizing the spine, and reducing pain. 

At Lineage Cell Therapeutics (Lineage), our mission is to pioneer a new branch of medicine, based on the transplant of fully-functional “replacement” cells for people impacted by serious conditions, including SCI. Lineage is developing OPC1, an investigational cell transplant, for the treatment of SCI. OPC1 is comprised of a population of neuronal cells which are intended to replace and restore the function of the cells which were damaged in SCI. Specifically, OPC1 contains oligodendrocyte progenitor cells (OPCs), which are naturally-occurring precursors to the cells which provide electrical insulation to nerve axons in the form of a myelin sheath.

Cell transplants like OPC1, offer patients the opportunity to receive healthy replacement cells (grown in a laboratory) to replace their existing cells that may be dysfunctional, damaged, or destroyed, such as in the case of persons affected by spinal cord injuries.

The OPC1 transplant is designed to replace or support cells that are absent or dysfunctional due to traumatic injury, with a goal to help improve the quality of life and restore or augment functional activity in persons suffering from traumatic cervical or thoracic SCIs, through a combination of possible mechanisms.

To date, 5 patients with thoracic spinal cord injuries and 25 patients with cervical spinal cord injuries have been enrolled in clinical trials of OPC1, with results from both studies published in the Journal of Neurosurgery Spine. The clinical development of OPC1 has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine.

In a phase 1 clinical trial of OPC1 for the treatment of thoracic SCI, all subjects were followed for at least 10 years: there were no unexpected serious adverse events attributable to the OPC1 cell transplant and no evidence of neurological decline, enlarging masses, further spinal cord damage or syrinx formation. In a Phase 1/2a clinical trial of OPC1 for the treatment of cervical SCI, all subjects were evaluated for at least 2 years: there were no unexpected serious adverse events related to the OPC1 cell transplant, no enrolled patients had worsening of neurological function and there was an indication of functional benefit compared to best available matched control (32% gained two or more levels of function within 12 months based on ISNCSCI scores).