StemCells, Inc. announced on December 15, 2001 that the first cohort of the Company’s Phase I/II clinical trial in chronic spinal cord injury have been successfully transplanted with the Company’s proprietary HuCNS-SC neural stem cells. This is a landmark clinical trial that has a unique design. What makes this clinical trial unique is the implantation of patients with progressively decreasing severity of spinal cord injury that are treated three sequential cohorts. The first cohort of patients all have spinal cord injury classified as AIS A, which is the most severe type of spinal cord injury as defined by the American Spinal Injury Association Impairment Scale or AIS.
Stephen Huhn MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. made this statement: “We are extremely pleased with our progress in this innovative trial. Having completed dosing of the AIS A cohort, screening for AIS B patients, who have a less severe, incomplete type of spinal cord injury, can now begin. Of course, our first priority is to assess safety in each patient, but we will also be evaluating trial patients for changes in sensation, motor and bowel/bladder function.”
Martin McGlynn, President and CEO of StemCells Inc added, “I am also pleased to announce that, in consultation with the clinical team at Balgrist Hospital (University of Zurich), the Company has decided to open enrollment for the remainder of the trial to patients living in the United States and Canada. We have received a large number of inquiries from patients in both countries, and hopefully this decision will come as good news to the spinal cord injury community, who were greatly disappointed by Geron’s recent decision to discontinue its spinal cord injury trial. We remain optimistic about the prospect of being able to demonstrate safety and clinical utility of our cells in this devastating condition, and are committed to funding our spinal cord injury program until such time as we can come up with a definitive outcome.”
The Phase I/II clinical trial of StemCells, Inc.’s HuCNS-SC purified human adult neural stem cells is designed to assess both safety and preliminary efficacy of this cell line in the treatment of spinal cord injuries. Twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level are planned for enrollment. The first three patients all have injuries classified as AIS A, in which there is no apparent neurological function below the level of spinal cord injury. The planned second and third cohorts will consist of patients who have spinal cord injuries classified as AIS B and AIS C, which are less severe than AIS A spinal cord injuries and show at least some preservation of sensory or motor function. This trial will assess safety of the cell line and treatment efficacy based on specific criteria. These clinical criteria include changes in sensation, motor and bowel/bladder function. Prior to implanting the next cohort, data from previous cohorts will be reviewed by an independent Data Safety Monitoring Committee.
All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord and will receive initial doses of medicines that suppress the immune system to ensure that the implanted cells do not elicit inflammation or are instantly rejected by the immune system. However, these drug treatments are temporary, since the central nervous system is surrounded by a blood-brain barrier than prevents the immune system from rejecting transplantations into the central nervous system. Temporary treatment with immunosuppressive drugs are necessary at the beginning of the procedure because the implantations breach the blood brain barrier and until this breach heals, the immune system has access to the implantation, but after the breach heals, immunosuppression is no longer necessary. Implanted patients will be evaluated regularly in the period after the transplant in order to monitor and assess the safety of the HuCNS-SC cells and the implantation procedure, and to determine if there are any neurological changes in the patients. The Company intends to follow the effects of this therapy long-term, and a separate four-year observational study will be initiated at the conclusion of this trial.
The trial is being conducted at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation. This institution has a global reputation for providing some of the highest quality examinations, treatments and rehabilitation opportunities to patients with serious musculoskeletal conditions. The clinic owes its leading international reputation to its unique combination of specialized medical services. The hospital’s carefully balanced, interdisciplinary network brings together under one roof medical specialties including orthopedics, paraplegiology, radiology, anesthesiology, rheumatology, and physical medicine.