Highlights from the 1st Spinal Cord Injury Investor Symposium: Cell Transplant Approaches

Spinal cord injury (SCI) affects an estimated 17,500 individuals each year in the United States, and less than 1% of those with SCI will experience complete neurologic recovery upon hospital discharge. Rehabilitation, assistive devices, medications, and surgery only provide benefit in mitigating symptoms, improving mobility, stabilizing the spine, and reducing pain.

In June, the first ever Annual SCI Investor Symposium was held in San Diego, California as a multidisciplinary conference where leaders from biotechnology companies and community advocacy organizations as well as patients with SCI came together to discuss their work, experiences, and the future of SCI treatment.

The Practical Neurology team sat down with Brian Culley, Chief Executive Officer of Lineage Cell Therapeutics (Carlsbad, CA), one of the 2 founding organizations for the symposium, to discuss the current landscape of SCI treatment, research, and their work in this field. Lineage Cell Therapeutics is a biotechnology company whose cell transplant approach 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 living with traumatic spinal cord injuries.

• The company has reported success in transplanting differentiated stem cells directly to the site of damage in thoracic and cervical SCI under its investigational new drug (IND) license with the Food and Drug Administration (FDA). So far, the company has reported on 2 clinical trials with its OPC1 clinical program: a safety study in 5 individuals with thoracic injury with 10 years of follow up (NCT01217008) and a 2-year dose escalation study in 25 individuals with cervical injuries (NCT02302157), both of which are published in the Journal of Neurosurgery Spine.
• In the 2-year dose escalation study, after 1 year of follow up, the 22 participants in the intent-to-treat population (ITT) had a mean upper extremity motor score (UEMS) improvement of 8.9 points per the International Standards for Neurological Classification of SCI (ISNCSCI). Seven ITT participants (32%) showed 2 motor levels of improvement and 21 (96%) showed at least 1 motor level of improvement. After 3 years, one participant showed an improvement of 3 motor levels.
• There were no unexpected serious adverse events attributable to the OPC1 cell transplant in either study.
• Two innovations which Lineage plans to introduce into its OPC1 clinical program include 1) improvements in purity of cell manufacturing, ensuring that all implanted cells are differentiated and of neural origin, which increases safety and potential benefit in patients, and 2) an enhanced spinal cord delivery method. In prior clinical trials, the procedure required immobilization of the patient with a large scaffold apparatus, necessitating disconnection from respiration equipment.
• Lineage’s next steps for the OPC1 program is a planned return to the clinic with a safety study in subacute and chronic SCI patients to test a novel delivery a device, which is attached directly to the patient with a set of retractors, enabling delivery of OPC1 cells while the patient is still breathing.

"When I sit down with the individuals who were on the clinical trial themselves, who maybe gained 2 levels of motor function or 3 levels of motor function on the gold standard assessment tools, it is incredible how much more independence and quality of life they have gained," said Brian Culley, Chief Executive Officer of Lineage Cell Therapeutics.