NEWARK, Calif.—StemCells Inc. has launched its Pathway Study, a Phase 2 proof-of-concept clinical trial using its proprietary HuCNS-SC platform of human neural stem cells, for the treatment of cervical spinal cord injury (SCI). This is the company’s first study accessing the efficacy of neural stem cells for the treatment of paraplegics dependent on wheelchairs and breathing tubes.
StemCells’ Pathway Study is reportedly the first clinical study designed to evaluate both the safety and efficacy of transplanting stem cells into patients with traumatic injury to the cervical spinal cord.
“The expansion of this trial into patients with cervical injury is exciting because even a gain of one to two segments in cervical spinal cord injury patients can allow for additional function in the upper extremities,” Greg Schiffman, chief financial officer of StemCells, tells DDNews.
The decision to pursue a therapy for spinal cord injury “was based on the large unmet medical need combined with the strength of the preclinical science supporting the use of our HuCNS-SC cells to treat victims of spinal cord injury,” Schiffman said. “We showed the cells could repair and replace damaged or lost cells such as the myelinating oligodendrocytes or new neurons.”
Approximately 1.3 million people in the U.S. report being paralyzed due to an SCI, and there currently are no effective treatments available. Approximately 56 percent of the spinal cord injuries occur in the cervical region. Overall, approximately 13 percent of SCI patients have no mobility, and 35 percent have limited mobility after the traumatic injury.
The upcoming 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. 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 patients for one year from the time of enrollment.
“StemCells Inc. has been evaluating our proprietary human neural stem cells (HuCNS-SC) for the treatment of spinal cord injury for over 10 years,” Schiffman says. “Our first preclinical work was published in the Proceedings of the National Academy of Sciences in 2005, demonstrating that our cells could promote locomotor recovery in spinal cord-injured mice.”
“Our first clinical trial in spinal cord injury was initiated in 2011 for victims with thoracic injuries to their spinal cord,” Schiffman adds. “The thoracic cord is responsible for sensory function. Earlier this year, the company completed enrollment and reported interim results from this trial on eight patients with at least six months of follow-up post transplantation. Half of the patients transplanted had significant post-transplant gains in sensory function. The interim results also continue to confirm the favorable safety profile of the cells and the surgical procedure.”
“The key to its success so far has been the HuCNS-SC product candidate,” he continues.
“We have conducted four clinical trials so far in disorders involving the brain, eye and spine. We have seen results consistent with our preclinical models in all of these studies. We believe that we have a platform in our HuCNS-SC human neural stem cells that has the ability to address a broad number of indications in the CNS, including spinal cord injuries. We see our HuCNS-SC platform as a next generation of cellular therapy and find that the vast majority of people seem to support the idea of using cells to treat serious disorders that have no other treatment options available.”
Schiffman believes that the cell therapy field is at a point “where clinical data is being generated and I think we will see several breakthrough therapeutic approaches validated over the next three to five years. This is a time of excitement and promise for the field of regenerative medicine, and I look forward to a time where we have several breakthrough stem cell-based therapies approved to treat serious disorders where there are no treatments available today.”
“The initiation of the Pathway Study represents a major milestone for StemCells Inc. as we pursue the development of a truly breakthrough therapy for spinal cord injury,” said Martin McGlynn, president and CEO of StemCells. “While we are thrilled by the prospect that patients with thoracic level injuries might be able to regain lost sensory function below the site of the injury, the possibility that patients with injuries to the cervical region of the cord might regain or improve lost motor function could be truly life-changing.”