StemCells, Inc., a world leader in the research and development of cell-based therapeutics for the treatment of CNS diseases and disorders, has enrolled its first subject in Cohort 2 of its phase II Pathway Study.
The study is designed to assess the efficacy of the company's proprietary HuCNS-SC platform technology (purified human neural stem cells) for the treatment of cervical spinal cord injury. Cohort 2 will enroll 40 patients and forms the single-blinded controlled arm of the phase II study. The primary efficacy outcome being tested in Cohort 2 is the change in motor strength of the various muscle groups in the upper extremities innervated by the cervical spinal cord.
The Pathway Study is the first clinical trial designed to evaluate both the safety and efficacy of human neural stem cells transplanted into the spinal cord of patients with cervical spinal cord injury. Traumatic injuries to the neck can damage the cervical spinal cord and result in impaired sensation and motor function of the arms, legs, and trunk, also referred to as quadriplegia. The trial has 3 cohorts. The primary Cohort is Cohort 2 which is being conducted as a randomized, controlled, single-blind Cohort and efficacy will be primarily measured by assessing motor function according to the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI). The trial will follow the participants for one year and will enroll up to 52 subjects.
Cohort 1 of the Pathway Study is an open-label, HuCNS-SC dose-escalation arm involving six patients. Safety data from all six subjects was reviewed by an independent Data Monitoring Committee and approval was provided to commence with Cohort 2. No safety or tolerability issues were seen at any of the dosing levels. The six-month outcome from Cohort 1 will be disclosed as interim data later this year.
Cohort 3 is an optional open label Cohort targeted to enroll 6 patients. This Cohort is designed to assess safety and preliminary efficacy in patients with less severe injuries (AIS C).
"The initiation of Cohort 2 begins the next phase of our clinical efforts towards a potential breakthrough therapy for spinal cord injury," said Stephen Huhn, M.D., FACS, FAAP, vice president, clinical research and chief medical officer at StemCells, Inc. "This is the first blinded, controlled clinical trial to be conducted using human neural stem cells. The goal of this proof-of-concept study is to demonstrate the potential efficacy of our cells as a treatment for victims of spinal cord injury. We currently have seven sites enrolling patients and expect to reach a total of fourteen active North American sites by year end. Conducting a multi-center study on this scale should allow us to efficiently enroll the study."
The company completed enrollment and dosing in its open-label phase I/II study in thoracic spinal cord injury in April 2014 and has reported top-line results. Sustained post-transplant gains in sensory function were demonstrated in seven of the twelve patients. Two patients in the phase I/II study converted from a complete injury (AIS A) to an incomplete injury (AIS B). The final results also continue to confirm the favorable safety profile of the cells and the surgical procedure.
The company's phase II Pathway 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. 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 and was designed to establish the cell dose for onward testing in the second cohort of the study.
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 in humans, 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.
StemCells, Inc. is currently engaged in clinical development of its HuCNS-SC platform technology (purified human neural stem cells) as a potential treatment for disorders of the central nervous system (CNS).