Lineage Marks First Patient Dosing in Study of Spinal Cord Injury Therapeutic

The first chronic spinal cord injury patient has been treated in Lineage Cell Therapeutics Inc.’s study of Oligodendrocyte Progenitor Cells (OPCs) for spinal cord injury treatment.

Occurring at UC San Diego Health, the DOSED (Delivery of Oligodendrocyte Progenitor Cells (OPCs) for Spinal Cord Injury: Evaluation of a Novel Device) clinical study aims to evaluate the safety and utility of a new parenchymal spinal delivery system, a novel delivery device developed to deliver OPC1 directly to the injury site in spinal cord injury (SCI) patients. The trial will enroll both subacute (between 21 to 42 days after injury) and chronic (between one and five years post-injury) SCI patients.

OPC1 is an investigational, allogeneic stem cell-derived cell transplant comprised of oligodendrocyte progenitor cells. OPC1 is designed to replace or support cells in the injured spinal cord that are absent or dysfunctional due to traumatic injury and is intended to help restore or augment functional activity in people suffering from an SCI. Improved functional activity can lead to greater mobility and enhanced quality of life for patients and significant cost-savings for caregivers. The first patient treated in DOSED was a neurologically complete SCI injury (American Spinal Injury Association Impairment Scale [AIS] grade A), with a single neurological level of injury (NLI) from T1 to T10, and the novel spinal delivery system successfully administered the intended one-time injection of 10 million OPC1 cells.

“Differentiated cell transplantation is a promising therapeutic approach that is increasingly being validated in a wide range of diseases and conditions. For this reason, it is exciting to advance the OPC1 program into further clinical testing and expand the OPC1-treated population to include chronic SCI patients,” Lineage Cell Therapeutics CEO Brian M. Culley said. “DOSED is the third clinical study of OPC1 and is evaluating a superior delivery system, designed to deliver our proprietary cells over several minutes and without the need for stopping patient ventilation during administration. The delivery system is also compatible with a forthcoming immediate-use thaw-and-inject formulation of OPC1 that we developed for this program, which will eliminate the lengthy dose preparation steps conducted in prior studies. This is the first time OPC1 has been administered to a patient with a chronic spinal cord injury, which is an important milestone because those patients represent an additional and larger potential treatable population for this experimental therapy. In addition to evaluating the safety and performance of the new delivery device, we also will be collecting functional assessments on all patients, which gives us the opportunity to investigate any signals of efficacy that may arise in these patients. We are pleased the first-ever use of this custom delivery solution had a successful outcome with no administration issues and look forward to opening this study to additional sites.”

OPC1 has extensive long-term safety data from two prior clinical trials: a five-patient Phase 1 safety study in acute thoracic SCI, where all active subjects have been followed for at least 13 years; and a 25-patient Phase 1/2a multicenter dose-escalation trial in subacute cervical SCI, where all active subjects have been evaluated for at least seven years. Long-term safety monitoring is ongoing for both studies, with no unexpected serious adverse events attributable to the OPC1 transplant being reported to date. Results from both studies have been published in the Journal of Neurosurgery: Spine. The OPC1 program was one of the first cell therapy clinical trials to be supported by the California Institute for Regenerative Medicine (CIRM) under Proposition 71. A publication focused on outlining the magnetic resonance imaging (MRI) evidence from the 25-patient Phase 1/2a multicenter dose-escalation trial of OPC1 in subacute cervical SCI is also forthcoming.

Lineage founded the Annual Spinal Cord Injury Investor Symposium in 2023 and has co-sponsored the event in partnership with The Christopher & Dana Reeve Foundation in each year since then. The goals of this collaborative effort include increasing disease awareness, improving the probability of success in product development, and supporting clinical trial participation. The Reeve Foundation is dedicated to curing spinal cord injury by funding research and improving the quality of life for individuals and families impacted by paralysis. Presenting companies have included AbbVie, Mitsubishi Tanabe, Neuralink, NervGen Pharma, Neuvotion, NovaGo Therapeutics, ONWARD, Paradromics, and Synchron.

OPC1 is an oligodendrocyte progenitor cell (OPC) transplant therapy designed to provide clinically meaningful recovery in, and improvements to, motor function in individuals with SCIs. OPCs are naturally occurring precursors to the cells that provide electrical insulation for nerve axons in the form of a myelin sheath. SCI most often occurs when the spinal cord is subjected to a severe crush or contusion injury and typically results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. There are approximately 18,000 new SCIs in the United States annually and more than 300,000 patients in total living with SCIs. There currently are no U.S. Food and Drug Administration (FDA)-approved drugs or interventions specifically for SCI treatment. OPC1’s clinical development has been partially funded by a $14.3 million grant from CIRM. OPC1 has received Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug designation from the FDA.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing allogeneic, or “off the shelf” cell therapies for serious neurological and ophthalmic conditions. Lineage’s programs are based on its proprietary cell-based technology platform and associated development and manufacturing capabilities. From this platform, Lineage designs, develops, manufactures, and tests specialized human cells with anatomical and physiological functions similar or identical to cells found naturally in the human body. These cells are created by applying directed differentiation protocols to established, well-characterized, and self-renewing pluripotent cell lines. These protocols generate cells with characteristics associated with specific and desired developmental lineages. Cells derived from such lineages are transplanted into patients in an effort to replace or support cells that are absent or dysfunctional due to degenerative disease, aging, or traumatic injury, and to restore or augment the patient’s functional activity.