First patient treated in Phase I/IIa regenerative medicine clinical trial for complete cervical spinal cord injury
Asterias Biotherapeutics, Inc. (CA, USA), a biotechnology company focused on developing regenerative medicine therapies, has commenced a Phase 1/2a trial evaluating escalating doses of AST-OPC1 in newly injured patients with sensory and motor complete cervical spinal cord injury.
At present there are no therapies or devices approved by the US FDA that can restore any function in individuals with recent spinal cord injuries (SCIs), despite over 12,000 people sustaining a SCI annually. Asterias Biotherapeutics, Inc. (CA, USA) is carrying out a registration program for AST-OPC1 (oligodendrocyte progenitor cells), which has neurologically complete cervical SCI as the first targeted indication.
After the Phase I trial demonstrated that a low dose of AST-OPC1 (2 million cells) was safe and well tolerated in five patients with neurologically complete, thoracic SCI, the first patient taking part in the Phase I/IIa trial (NCT02302157) has now been treated. Dr Donald Peck Leslie, medical director of Shepherd Center and principal investigator for this study site, commented: “If AST-OPC1 could deliver even modest improvements in motor or sensory function, it would result in significant improvements in quality of life for people with SCI.”
The trial, an open-label, single-arm study, will be conducted at up to eight centers in the USA. It will investigate three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells, 14–30 days post injury. It will be administered to 13 patients with subacute, C5 to C7, neurologically complete cervical SCI: individuals with no sensation and movement below their injury site, with severe paralysis of the upper and lower limbs. Neurological exams and imaging methods will be applied to assess the safety and activity of the cells.
If the first two cohorts of this study achieve the safety targets, Asterias will seek approval to expand enrollment from 13 patients to up to 40, to increase the statistical confidence of the safety and efficacy results, decrease the risks of the AST-OPC1 program and increase the product’s potential for accelerated regulatory approval.
“The commencement of dosing in this Phase I/IIa trial is a significant advancement of our AST-OPC1 development program in that this study is specifically designed to evaluate the product at the doses and in the population where it has the maximum potential to bring benefit to patients,” stated President and CEO of Asterias, Pedro Lichtinger. “Individuals with SCI have severe disabilities that can significantly shorten projected lifespan, impact quality of life and result in lifetime costs of care of US$3–4 million. We are grateful for the interest of patients with SCI to participate in this program.”
Asterias received a grant from the California Institute for Regenerative Medicine (CIRM) providing $14.3 million of nondilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.