Spinal cord injury is a devastating disease with no available treatments. It disproportionately affects young people who then lives many decades with the debilitation. In this project, we propose to develop a human neural stem cell line to treat spinal cord injury patients within 14 days after the injury. Administration of the cells into the injured tissue will limit the tissue from further damage and provide replacement neurons and glia that will promote self-repair and regeneration for the rest of the patient's life. During this project, we will assess the therapeutic as well as any toxicity potentials of the cells, using rats and pigs that will be injured in a manner similar to human spinal cord injury. These animal models will provide a rigorous testing grounds to prove or disprove potential clinical usefulness of the cells. If the cells can successfully treat rats or pigs with no major toxicity, then we will submit a IND application to the FDA to conduct a Phase 1 clinical trial with spinal cord injured patients.
The proposed research is to develop a stem cell therapy to treat spinal cord injury patients in the acute phase after the initial injury event. By intervening early, benefits of the stem cell transplantation to the patients are likely to be greatest. Thus, the patients in the trials will require intensive care and thus only the local population within California could participate. The treatment requires surgery and invasive injection of the cells into the injury site, which will require a special training. Thus, the benefit to the State of California is first access to this therapy for about 2-3 years before it becomes available elsewhere. Secondly, California has two federally funded Regional Spinal Cord Injury Care Centers that research various aspects of rehabilitation after spinal cord injury. Conducting clinical trials in California will become further catalysts to strengthen statewide treatment and rehabilitation centers for spinal cord injury patients.