StemCells, Inc. announced that is has filed an application with Swiss-Medic, the Swiss regulatory agency for therapeutic products, to conduct a clinical trial in Switzerland of the company's HuCNS-SC purified human neural stem cells in chronic spinal cord injury patients. If authorized, the study would enroll patients who are three to 12 months post-injury.
“With this filing we have taken a tangible step to broaden the clinical development of our HuCNS-SC product candidate beyond the brain and into the spinal cord,” said Martin McGlynn, president and CEO of StemCells, Inc. “This exciting initiative is supported by extensive preclinical research demonstrating the ability of our proprietary cells to restore lost motor function when transplanted in the chronic spinal cord injury setting. The prospect of extending the treatment window to months or longer following injury would mean that a much larger population of injured patients could potentially benefit from such an approach” he added.
Stephen Huhn, MD, FACS, FAAP, vice president and Head of the CNS Programme at StemCells, Inc., added, “Switzerland is home to some of the leading clinicians and medical centers in the world engaged in the treatment and rehabilitation of spinal cord injury patients, which makes it an excellent setting for this type of trial. While we have yet to secure Swiss-medic authorization to initiate the study, we are pleased to have already received approval from the respective ethics committees at our anticipated trial site and the local Canton, both of which are prerequisites for making a submission of this kind in Switzerland.”
The results of numerous collaborative preclinical studies with researchers at the University of California, Irvine (UCI) demonstrate the significant therapeutic potential of the Company's human neural stem cells for the treatment of spinal cord injury. Data published in several peer-reviewed journals show that the cells engraft, migrate along the spinal cord to the point of injury, and then differentiate into neurons and specialized cells called oligodendrocytes that create the insulation (myelin) necessary for proper transmission of nerve impulses from the brain to below the level of injury.
When transplanted in spinal cord-injured mice at both sub-acute and chronic injury time points, the cells have been shown to form protective myelin sheaths around damaged nerve axons and enable a significant and persistent recovery of walking ability.
Aileen Anderson, PhD, Associate Professor in the Departments of Physical Medicine and Rehabilitation, and Anatomy and Neurobiology at UCI, commented, “The preclinical data we have seen to date provide a compelling rationale for advancing these neural stem cells into clinical testing for chronic spinal cord injury. Restoring some degree of function for patients at later time points beyond the acute injury phase could have a transformative impact on the field as there are no effective treatment options for them today. The demonstrated ability of these cells to repair an injured spinal cord and promote functional motor recovery in animals could translate into improved quality of life for individuals living with paralysis, and I am excited to see StemCells take this important next step toward what I hope will one day become a viable therapy for a broad population of spinal cord-injured patients.”
According to a recent study initiated by the Christopher and Dana Reeve Foundation, nearly 1.3 million people in the US alone are estimated to be living with chronic spinal cord injury. Chronic spinal cord injury is characterized as a point in time after injury in which inflammation has stabilized and behavioral recovery has reached a plateau. In spinal cord-injured patients, the chronic phase typically does not set in until several weeks or months following the injury. To date, treatment approaches have generally targeted the acute and sub-acute time points post-injury, which are considered to be hours or days following injury. StemCells hopes to address a broader population of spinal cord-injured patients by opening the window of opportunity for therapeutic intervention to months or longer post-injury.
StemCells' lead product candidate, HuCNS-SC cells, is a highly purified composition of human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted in the central nervous system with no sign of tumour formation or adverse effects. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, this suggests the possibility of a durable clinical effect following a single transplantation.
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. In its therapeutic product development programs, StemCells is targeting disorders of the central nervous system and the liver. StemCells' lead product candidate, HuCNS-SC cells (purified human neural stem cells), is currently in clinical development for the treatment of two fatal neurodegenerative disorders in children, and in preclinical development for spinal cord injury and retinal disorders such as age-related macular degeneration.
StemCells also markets stem cell research products, including media and reagents, under the SC Proven brand, and is developing stem cell-based assay platforms for use in pharmaceutical research, drug discovery and drug development.