Scimitar Equity Blog

CUR filed an IND application with the FDA to begin a Phase I safety clinical trial for chronic spinal cord injury with its spinal cord stem cells. This multicenter Phase I safety trial will enroll a total of 16 long-term, or chronic, spinal cord injury patients, with an American Spinal Injury Association (ASIA) Grade A level of impairment, 1 to 2 years post-injury.  ASIA A refers to a patient with no motor or sensory function in the relevant segments and is considered to be complete paralysis.

Clinical studies with chronic SCI patients have demonstrated that:

• Even several years after motor- and sensory-complete SCI, intensive and task-specific rehabilitation can improve motor function as measured by the ASIA Impairment Scale (AIS),
• These clinical experiences, together with results from numerous animal studies of SCI, illustrate that even the complete, chronically-injured cord can undergo some degree of recovery and reorganization,
• Transplantation of CUR’s human spinal stem cells (HSSC) is meant to provide a neuron-rich substrate to the injured segments of a patient’s spinal cord to promote further repair, regeneration, and reorganization,
• The goal is to harness this inherent plasticity and promote reorganization by combining stem cell transplantation with the modern concept of activity-guided rehabilitation.

Currently, no predictive animal model of chronic human SCI exists:

• However, in a rat model of ischemia-induced spinal cord injury that generates the condition of permanent paraplegia, transplantation of CUR’s HSSC into the injury site leads to significant motor recovery (reported in NEUROSCIENCE, 6/29/07, in conjunction with the University of California at San Diego),
• Detailed analysis revealed robust graft survival, extensive neuronal differentiation, and integration of grafted cells into the host circuitry, all outcomes which we believe are essential for recovery of function in human patients,
• Similar results were obtained by CUR in rodent models of ALS in which transplanted HSSC again showed extensive neuronal differentiation and integration with host neuronal circuitry, and led to the rescue of at-risk motor neuron populations (reported in Transplantation, 10/06, and Journal of Comparative Neurology, 3/09, in conjunction with Johns Hopkins University),
• These studies further demonstrated that CUR’s HSSC transplants secreted important neuroprotective agents and extended long-distance axons into the host.

CUR believes that, in chronic SCI; HSSC transplants may promote reorganization of segmental circuitry over the long-term.

• In the cervical region of the spinal cord, this could result in improved breathing capacity and recovery of sensori-motor functions of the upper limbs,
•  Segmental reorganization induced by, and utilizing graft-derived neurons, may also result in improved locomotion,
• Neurons differentiated from CUR’s HSSC grafts in chronic thoracic injuries may serve as a bridge to connect the axons located above the site of injury to neurons of segments below the injury site,
• HSSC grafts may also encourage axons to regenerate through the graft to segments below the injury.

There are more than 10,000 new spinal cord injuries (SCI) in the US each year.  At the time of a recent survey done by the Christopher & Dana Reeve Foundation, there were approximately 840,000 people living with chronic SCI. According to the National Spinal Cord Injury Association, 85% of SCI patients who survive the first 24 hours are still alive 10 years later.