Lineage Cell Therapeutics (NYSE American and TASE: LCTX) has launched a groundbreaking clinical study that could potentially transform the treatment landscape for spinal cord injury (SCI) patients. The DOSED study (Delivery of Oligodendrocyte Progenitor Cells for Spinal Cord Injury: Evaluation of a Novel Device) will evaluate both the safety and utility of a novel delivery system for their investigational cell therapy, OPC1.
Novel Delivery System and Study Design
The study will utilize the Manual Inject Parenchymal Spinal Delivery System (MI PSD System), specifically developed to deliver OPC1 cells directly to injury sites. This innovative system allows for cell administration over several minutes without interrupting patient ventilation, representing a significant technical advancement in cell therapy delivery.
The trial will enroll two distinct patient populations:
- Subacute patients (21-42 days post-injury)
- Chronic patients (1-5 years post-injury)
"Differentiated cell transplantation is a promising therapeutic approach," stated Brian M. Culley, Lineage's CEO. "The inclusion of chronic SCI patients represents a significant milestone, as it opens up a larger potential patient population for this experimental therapy."
Promising Safety Profile and Previous Clinical Experience
OPC1's development is supported by extensive safety data from two previous clinical trials:
- A Phase 1 safety trial in acute thoracic SCI with 13+ years of follow-up
- A 25-patient Phase 1/2a multicenter trial in subacute cervical SCI with 7+ years of follow-up
Both studies have reported no unexpected serious adverse events attributable to OPC1 transplantation, with results published in the Journal of Neurosurgery: Spine.
Therapeutic Mechanism and Market Potential
OPC1 consists of oligodendrocyte progenitor and related glial cells designed to replace or support damaged spinal cord cells. These cells are crucial for providing electrical insulation for nerve axons through myelin sheath formation. The therapy aims to restore or enhance functional activity in SCI patients, potentially improving mobility and quality of life while reducing caregiver burden.
The market need is substantial, with approximately 18,000 new spinal cord injuries occurring annually in the U.S. and over 300,000 patients living with SCIs. Currently, there are no FDA-approved treatments specifically for SCI.
Study Endpoints and Monitoring
The DOSED study will evaluate multiple parameters:
Primary endpoint:
- Safety assessment of the delivery system through adverse event monitoring over 30 days
Secondary endpoints:
- Adverse event monitoring through 90 days
- Assessment of immunosuppression effects
Exploratory endpoints include:
- Changes in neurological function
- Pain assessment
- Quality of life measurements
- Patient and clinician global impressions of change
The first study site will be UC San Diego Health, with the potential for additional centers to join the trial.
Regulatory Support and Funding
The OPC1 program has received significant recognition and support:
- Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA
- Orphan Drug designation
- $14.3 million grant from the California Institute for Regenerative Medicine (CIRM)
The program has also garnered support from The Christopher & Dana Reeve Foundation, which has partnered with Lineage for the Annual Spinal Cord Injury Investor Symposium, demonstrating the broader ecosystem of support for advancing SCI treatments.
