Date: 2015-05-14
Type of information: Presentation of results at a congress
phase: 1-2
Announcement: presentation of results at the 4th Joint International Spinal Cord Society (ISCoS) and American Spinal Injury Association (ASIA) meeting being held in Montreal, Canada.
Company: StemCells (USA - CA)
Product: HuCNS-SC human neural stem cells
Action
mechanism: stem cell therapy
Disease: thoracic spinal cord injury
Therapeutic area: Rare diseases - Regenerative medicine
Country: USA
Trial
details: The Pathway Phase II study, titled \"Study of Human Central Nervous System (CNS) Stem Cell Transplantation in Cervical Spinal Cord Injury,\" will evaluate the safety and efficacy of transplanting the Company\'s proprietary human neural stem cells (HuCNS-SC cells), into patients with traumatic injury in the cervical region of the spinal cord. Conducted as a randomized, controlled, single-blind study, the trial will measure efficacy by assessing motor function according to the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI). The primary efficacy outcome will focus on change in upper extremity strength as measured in the hands, arms and shoulders. The trial will enroll approximately 52 subjects and follow the patients for 12 months post-transplant. The first cohort of six patients completed enrollment in April. This is a dose confirmation cohort. The results from this cohort will determine the cell dosage to be used for the remainder of the study. The six-month interim data from this cohort will be disclosed later this year. All subjects who enrolled in the trial received HuCNS-SC cells through direct transplantation into the spinal cord and underwent temporary treatment with immunosuppressive drugs. Evaluations were performed regularly throughout the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any change in neurological function. Preliminary efficacy was evaluated based on defined clinical endpoints using established spinal cord injury scales assessing changes in sensory and motor function. Patients were enrolled in a separate observational study after completing the twelve-month Phase I/II trial. Enrollment included seven AIS A and 5 AIS B patients. The study showed a favorable safety, feasibility and tolerability profile with no adverse events associated to the HuCNS-SC cells. Twelve months after transplantation of the HuCNS-SC cells, data showed gains in sensory function in three of the seven AIS A patients and four of the five AIS B patients. In addition, two patients progressed from the most severe classification, AIS A, to a lesser degree of injury grade, AIS B. (NCT02163876)
Latest
news: * On May 14, 2015, StemCells, a leading stem cell company developing novel cell-based therapeutics for treating diseases of the central nervous system, announced that Dr. Armin Curt, principal investigator, will present a summary of the safety and preliminary efficacy data from the Phase I/II study investigating Human Central Nervous System Stem Cell (HuCNS-SC) intramedullary transplantation in thoracic spinal cord injury. The summary will be presented at the 4th Joint International Spinal Cord Society (ISCoS) and American Spinal Injury Association (ASIA) meeting being held in Montreal, Canada. The international, open-label, Phase I/II trial evaluated both safety and preliminary efficacy of StemCells, Inc.\'s proprietary HuCNS-SC human neural stem cells as a treatment for chronic spinal cord injury. The trial enrolled twelve patients who had suffered injury to the thoracic cord and were in the early chronic stage of recovery. The severity of spinal cord trauma is classified by the ASIA Impairment Scale (AIS) according to the degree of sensory and motor loss. Patients classified as AIS A, the most severe injury, have lost both sensory and motor function below the level of injury. AIS B patients are considered less severely injured because, although they also have no motor function below the level of injury, some limited sensory function is maintained. The protocol was specifically designed to test safety and preliminary efficacy across this spectrum of injury severity, and ultimately the trial transplanted seven AIS A and five AIS B patients. The analysis of the study demonstrated that the surgical transplantation technique and cell dose were safe and well tolerated by all patients. HuCNS-SC cells were injected directly into the cord both above and below the level of injury and sequential examinations of the patients over the course of twelve months showed no abnormal changes in spinal cord function associated with the transplantation technique. There were no adverse events attributed to the HuCNS-SC cells. In addition to safety, analysis of the twelve-month data revealed sustained improvements in sensory function that emerged consistently around three months after transplantation and persisted until the end of the study. The patterns of sensory gains were confirmed to involve multiple sensory pathways and were observed more frequently in the patients with less severe injury; three of the seven AIS A patients and four of the five AIS B patients showed signs of positive sensory gains confirming the previously released interim results. In addition, two patients progressed during the study from the most severe classification, AIS A, to the lesser degree of injury grade, AIS B. In follow-up to the thoracic study, the Company has initiated a Phase II trial designed to measure efficacy in cervical spinal cord injury. The Phase II trial, also called the Pathway study, is a controlled study and was initiated in late 2014.
