Positive interim efficacy data have been reported from an ongoing phase 1/2a trial of AST-OPC1 (Asterias Biotherapeutics), an oligodendrocyte progenitor population derived from human embryonic stem cells, in patients with complete cervical spinal cord injury. While it is still early in the study, all of the patients have shown at least one motor level of improvement.
The response to treatment is being measured on the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) scale, which is widely used to quantify the functional status of patients with spinal cord injuries. As suggested by existing research, patients with complete cervical spinal cord injuries that show two motor levels of improvement on at least one side may regain the ability to perform daily activities, such as feeding, dressing, and bathing.
The efficacy target was recommended by the independent Spinal Cord Outcomes Partnership Endeavor (SCOPE) based on published data. SCOPE is affiliated with the American Spinal Injury Association.
In the ongoing study, cohort 1 consists of three patients who received a low dose of two million AST-OPC1 cells. The interim efficacy and safety results are as follows:
Cohort 2 consists of five patients who received 10 million AST-OPC1 cells. The interim efficacy and safety results are as follows:
Efficacy results at six months after the implantation of 10 million AST-OPC1 cells in patients with complete cervical spinal cord injury will be available in January 2017. The data will focus on improvements in physical functioning of the upper extremities (i.e., fingers, hands, and arms) of each treated patient, as indicated by the ISNCSCI scale.
Source: Asterias Biotherapeutics; September 14, 2016.