NEWARK, Calif., Dec. 18, 2014 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM), a world leader in the research and development of cell-based therapeutics for the treatment of disorders of the central nervous system, announced today that it has transplanted the first subject in its Phase II Pathway® Study assessing the efficacy of its proprietary HuCNS-SC® (purified human neural stem cells) platform technology for the treatment of cervical spinal cord injury (SCI). The transplant was performed at the University of Miami Hospital within the Miller School of Medicine, home to The Miami Project to Cure Paralysis, one of the world's most comprehensive spinal cord injury research centers dedicated to finding effective treatments for paralysis.
"The participant tolerated the procedure well and is recovering from the surgery as expected," said Allan D. Levi, M.D., Ph.D., F.A.C.S., Robert M. Buck Distinguished Chair in Neurological Surgery at the University of Miami Miller School of Medicine and Principal Investigator for the center. "Our center is a leader in clinical research aimed at curing paralysis, and we are excited to be participating in this breakthrough approach to spinal cord injury repair. The Pathway study is designed to measure the potential of these human neural stem cells, HuCNS-SC, as a possible treatment for repairing some aspects of spinal cord injury. Restoring or improving motor function would be life changing for these patients."
The Pathway Study is the first clinical trial designed to evaluate both the safety and efficacy of transplanting human neural stem cells into patients with cervical spinal cord injury. Traumatic injuries to the cervical (neck) region of the spinal cord, also known as tetraplegia or quadriplegia, impair sensation and motor function of the hands, arms, legs, and trunk. The trial will be conducted as a randomized, controlled, single-blind study and efficacy will be primarily measured 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 follow the participants for one year and will enroll up to 52 subjects.
"We are working with all due speed to pioneer a revolutionary therapeutic approach for victims of spinal cord injury, a debilitating condition that has significant unmet need and no effective treatment," said Stephen Huhn, M.D., FACS, FAAP, Vice President, Clinical Research and CMO at StemCells, Inc. "Enrollment of the first patient with cervical injury is an important step in our clinical development, which ultimately has the goal of improving motor function and restoring a level of independence for individuals living with chronic spinal cord injury. The Pathway Study is a natural evolution from our Phase I/II trial in thoracic spinal cord injury."
The Company completed enrollment and dosing in its open-label Phase I/II study in thoracic spinal cord injury in May 2014 and has reported interim results on all 12 subjects. Post-transplant gains in sensory function below the level of injury were demonstrated in half of the subjects. Two subjects converted from a complete injury (AIS A) to an incomplete injury (AIS B). The interim results also continue to confirm the favorable safety profile of the cells and the surgical procedure.
About the Pathway Spinal Cord Injury Clinical Trial
The StemCells, Inc. Pathway Phase II clinical trial, 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.
Information about the Company's spinal cord injury program can be found on the StemCells, Inc. website at:
Patient testimonials from the Phase I/II study can be found at:
http://www.stemcellsinc.com/News-Events/Video-Library.htm
Information for patients interested in participating in the study is available at the Pathway website at:
http://www.sciresearchstudy.com
Additional information about the clinical trial is available at:
http://clinicaltrials.gov/ct2/show/NCT02163876?term=stem+cells+cervical+spinal+cord+injury&rank=1
About HuCNS-SC Cells
StemCells, Inc. has demonstrated human safety data from completed and ongoing clinical studies in which its proprietary HuCNS-SC cells have been transplanted directly into all three components of the central nervous system: the brain, the spinal cord and the eye. StemCells, Inc. clinicians and scientists believe that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the CNS. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation. The HuCNS-SC platform technology is a highly purified composition of human neural stem cells (tissue-derived or "adult" stem cells). Manufactured under cGMP standards, the Company's HuCNS-SC cells are purified, expanded in culture, cryopreserved and then stored as banks of cells, ready to be made into individual patient doses when needed.
About StemCells, Inc.
StemCells, Inc. is currently engaged in clinical development of its HuCNS-SC (purified human neural stem cells) platform technology, as a potential treatment for diseases and disorders of the central nervous system. Interim data from the Company's Phase I/II clinical trial in thoracic spinal cord injury shows measurable gains involving multiple sensory modalities and segments in half of the subjects, two of whom converted from complete injury (AIS A) to incomplete injury (AIS B), post-transplant. Enrollment has recently commenced in the Company's Phase II clinical trial in cervical SCI. StemCells, Inc. has also completed enrollment and treatment in its Phase I/II clinical trial in geographic atrophy of age-related macular degeneration (GA-AMD), the most severe form of dry AMD, which is the leading cause of blindness in the elderly. Interim results for those subjects with 12 month follow-up post transplantation of HuCNS-SC cells into the eye, show a reduction in the rate of disease progression as compared to the control (untreated) eye and to the expected natural history of the disease. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company showed preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. Further information about StemCells, Inc. is available at http://www.stemcellsinc.com.
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding whether the improvements in sensory function seen in the Company's Phase I/II clinical study of spinal cord injury will persist and whether they will prove to be clinically meaningful; the prospect for evaluating trial patients for changes in their sensation, motor function and bowel/bladder function; the potential of the Company's HuCNS-SC cells to treat spinal cord injury and other central nervous system disorders; and the future business operations of the Company, including its ability to conduct clinical trials as well as its other research and product development efforts. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including the fact that additional trials will be required to demonstrate the safety and efficacy of the Company's HuCNS-SC cells for the treatment of any disease or disorder; uncertainty as to whether the FDA or other applicable regulatory agencies will permit the Company to continue clinical testing in spinal cord injury or in future clinical trials of proposed therapies for other diseases or conditions; uncertainties regarding the Company's ability to recruit the patients required to conduct its clinical trials or to obtain meaningful results; uncertainties regarding the Company's ability to manufacture viable cells sufficient to enroll the patients planned for the Company's Phase II studies; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current and planned research and development operations; uncertainty as to whether HuCNS-SC cells and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; and other factors that are described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2013, and in its subsequent reports on Form 10-Q and Form 8-K.