Asterias Biotherapeutics provided an update from its ongoing phase 1/2a clinical trial of a stem cell treatment for severe spinal cord injury.
Here are four things to know:
1. The SCiStar study involves three escalating doses of AST-OPC1 involves three escalating doses in 25 SCI patients. AST-OPC1 is an oligodendrocyte progenitor cell population derived from human embryonic stem cells.,
2. The study consists of five cohorts. Each subject from cohorts 3 and 4 have completed a 12-month follow-up.
3. The updated study results demonstrate:
- No serious adverse effects related to AST-OPC1 to date.
- Cell engraftment: Almost all — 92 percent — of cohort 3 and 4 subjects have MRI scans at 12 months consistent with the formation of a tissue matrix at the injury site. This indicates that AST-OPC1 cells have likely engrafted at the injury site and helped to prevent cavitation.
- Improved motor function: At 12 months, all cohort 3 subjects recovered at least one motor level on at least one side, compared to 83 percent of cohort 4 subjects.
4. Asterias Biotherapeutics plans to request a formal meeting with the FDA under the OPC1 program's Regenerative Medicine Advanced Therapy designation. The company expects to publish the 12-month data for the entire study in the first quarter of 2019 and apply for funding to support a randomized controlled trial for OPC1.
"The study data has been consistently positive and reaffirms our view that OPC1 is safe, that it durably engrafts and has the potential to improve motor function," said Asterias Biotherapeutics' CMO Ed Wirth, MD, PhD.