for Spinal Cord Injury
OPC1 is an oligodendrocyte progenitor cell therapy that has been cleared for evaluation in a device safety study, the DOSED study, in subacute and chronic spinal cord injury patients.
Oligodendrocyte progenitor cells (OPCs) are naturally-occurring precursors to the cells which provide electrical insulation for nerve axons in the form of a myelin sheath. SCI 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 and sexual function. Based on the multiple reparative properties associated with OPC1, we believe this candidate cell therapy product is ideally suited to treat neurological conditions such as SCI and other demyelination and demyelination disorders of the central nervous system.
On February 13, 2024, Lineage announced the clearance by the U.S. Food and Drug Administration (FDA) of its Investigational New Drug amendment (INDa) to enable the return to clinical testing for the OPC1 program. Pursuant to the INDa, Lineage has initiated activities to open its first clinical site in the DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) clinical study, to evaluate the safety and utility of a novel spinal cord delivery device in subacute and for the first time, chronic SCI patients.
OPC1 has received a regenerative medicine advanced therapy (RMAT) and Orphan Drug designations from the FDA, which include accelerated programs intended to facilitate the development and review process, including early interactions with FDA during clinical development and enhanced favorability for marketing approval.
In 2016, Jake Javier was paralyzed from the neck down. In late 2022, he is set to graduate from Duke University with his Master’s Degree in Biomedical Engineering, with plans to help those impacted by neurological injuries or diseases.
Highlighting the progress made by Kris Boesen and Lucas Lindner, both of whom received Lineage’s OPC1 cell therapy following traumatic spinal cord injury (SCI).
Learn More About Spinal Cord Injury and Lineage’s OPC1 Cell Therapy
There are approximately 18,000 new spinal cord injuries annually in the U.S. (NSCIC SCI Facts and Figures at a Glance (2019), and there are currently no drugs approved by the FDA specifically for the treatment of SCI.
OPC1 has been tested in two clinical trials to date; a five patient Phase 1 study in acute thoracic spinal cord injury, where all subjects were followed for at least 10 years, and a 25 patient Phase 1/2a study in subacute cervical spinal cord injury, where all subjects were evaluated for at least 2 years (the “SCiStar trial”). The clinical development of OPC1 has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine.
Expected Recovery1 vs OPC1: Motor Function Gains
In November 2019, we reported positive results from the ongoing SciStar study of OPC1, where the overall safety profile of OPC1 has remained excellent with robust motor recovery in upper extremities maintained through Year 2 patient follow-ups available to date. In July 2020, we reported that following the transfer of OPC1 manufacturing to our cGMP manufacturing facility in Israel, key process improvements have been developed and implemented. Lineage also strengthened its patent position in order to protect the OPC1 processes, product and composition, and methods of use. In December 2020, Lineage reported it has developed an enhanced differentiation process, leading to major improvements in production and quality of its OPC1 cell therapy drug product.
In February 2021, we announced that we had entered into an exclusive option and license agreement with Neurgain Technologies, Inc., a medical device company that is commercializing technology developed by neurosurgeons at the University of California San Diego. Under the terms of the agreement, the Company and Neurgain will collaborate on the clinical testing of Neurgain’s novel Parenchymal Delivery Injection (“PDI”) system, which is designed to allow for the administration of cells to the spinal cord without stopping the patient’s respiration. Elimination of the need to stop respiration during surgery is expected to reduce the complexity, risk, and variability of administering cells to the area of injury.
View our pipeline to see our other programs in development.
1. Steeves JD, Lammertse DP, Kramer JL, Kleitman N, Kalsi-Ryan S, Jones L, Curt A, Blight AR, Anderson KD. Outcome Measures for Acute/Subacute Cervical Sensorimotor Complete (AIS-A) Spinal Cord Injury During a Phase 2 Clinical Trial. Top Spinal Cord Inj Rehabil. 2012 Winter;18(1):1-14. doi: 10.1310/sci1801-1. Epub 2012 Jan 31. PMID: 23239927; PMCID: PMC3519288.
2. Fessler, R. G., Ehsanian, R., Liu, C. Y., Steinberg, G. K., Jones, L., Lebkowski, J. S., Wirth, E. D., III, & McKenna, S. L. (2022). A phase 1/2a dose-escalation study of oligodendrocyte progenitor cells in individuals with subacute cervical spinal cord injury, Journal of Neurosurgery: Spine (published online ahead of print 2022). Retrieved Aug 19, 2022, from https://thejns.org/spine/view/journals/j-neurosurg-spine/aop/article-10.3171-2022.5.SPINE22167/article-10.3171-2022.5.SPINE22167.xml