Lineage Cell to Evaluate Novel Delivery of OPC1 Cell Therapy for Spinal Cord Injury
The company expects to open the first clinical site in the DOSED study in the second quarter of 2024.
Richard Fessler, MD
Credit: Rush University Medical Center
The FDA has cleared Lineage Cell Therapeutics’ investigational new drug (IND) amendment for OPC1 cell therapy transplant for treating patients with spinal cord injury (SCI), allowing the company to initiate a study evaluating a novel delivery device for the therapy in patients with subacute and chronic SCI.
Lineage Cell has initiated activities to open its first clinical site in the new study evaluating the novel delivery device, termed DOSED (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device. The company expects to be able to open the initial in the second quarter of 2024, following trial preparations and a grant submission to the California Institute for Regenerative Medicine in the first quarter of the year.
“Lineage’s oligodendrocyte cell transplants are designed to replace or support cells that are absent or dysfunctional due to traumatic injury, with the goal of helping to improve the quality of life and restore or augment functional activity to persons suffering from traumatic cervical or thoracic spinal cord injuries. The clearance of our INDa and the initiation of OPC1 clinical
testing under our sponsorship represents a significant milestone for this program, and reflects our commitment to developing modern cell therapy product candidates,” Brian M. Culley, CEO, Lineage, said in a statement. “We are excited by the opportunity to build upon the promising results achieved with OPC1 in previous trials, and to continue to seek improvements in how our therapy is prepared and administered.”
OPC1 is an investigational allogeneic oligodendrocyte progenitor cell transplant therapy that has been evaluated in both acute thoracic and subacute cervical SCI. These indications were evaluated in a phase 1, 5-patient safety trial with follow-up of at least 10 years, and in a phase 1/2a, 25-patient, multicenter, dose-escalation clinical trial with follow-up of at least 2 years, respectively. In these trials, OPC1 demonstrated good safety and some signals of efficacy, with no serious adverse events (AEs) and quality of life improvements.
“Most people with spinal cord injury, at some point in their life, will have a fracture. And that's because their bones become demineralized. So, we're trying to figure out why, and what we can do to stop that, so that we can minimize fractures in these patients. So, they're living [with] normal life expectancy now, but we want to give them as good a quality of life as we potentially can,” primary investigator Richard Fessler, MD, professor of neurosurgery, Rush University Medical School, previously told CGTLive® about the OPC1 program.
DOSED will be an open-label, multi-center, device safety study that plans to enroll 3-5 patients each with subacute SCI and stable chronic complete or incomplete traumatic, focal SCI affecting either cervical (C4-C7) orthoracic (T1-T10) vertebrae. The study will primarily evaluate safety of the novel delivery device by measuring frequency and severity of related AEs through 30 days after OPC1 injection. Secondary endpoints include other safety and tolerability endpoints through 90 days of injection, such as AEs of special interest or AEs related to OPC1 or concomitant immunosuppression. Exploratory efficacy endpoints will evaluate neurological impairment, function, and pain; changes in International Standards for Neurological Classification of Spinal Cord Injury, SCIM, International Spinal Cord Injury Pain Questionnaire at 30, 90 and 365 days post-OPC1 injection; and patient and clinician impressions of changes in quality of life from baseline at 30, 90, and 365 days post-OPC1 injection.
“The DOSED clinical study in particular is intended to evaluate the safety and performance of a new delivery device, which is compatible with our forthcoming immediate-use formulation, and which does not require cessation of patient ventilation during administration. We believe these improvements can lead to a safer surgical procedure for surgeons and patients,” Culley added. “This study also will mark the first time that OPC1 is administered to chronic SCI patients, and we will be collecting efficacy assessments in addition to the primary outcome measures of safety. Completing this regulatory step also enables us to proceed with our planned CLIN-2 grant application to CIRM to request external financial support for the OPC1 program.”
