StemCells 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,” says Allan D Levi, Robert M Buck distinguished chair in neurological surgery at the University of Miami Miller School of Medicine and principal investigator for the centre. “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. The trial will be conducted as a randomised, 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 enrol up to 52 subjects.
“Enrolment 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,” says Stephen Huhn, vice president, clinical research and chief medical officer at StemCells.
The company completed enrolment 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 favourable safety profile of the cells and the surgical procedure.