|Asterias Biotherapeutics Receives FDA Clearance to Enroll C-4 Patients in SCiStar Study|
The protocol amendment will expand patient eligibility and enable study investigators to administer AST-OPC1 to patients with injuries at one vertebral level higher than the trial's previous C-5 limitation, to the fourth cervical vertebra down, known as C-4, near the middle of the neck. A C-4 cervical level injury, the second most common level of SCI in the SCiStar study's targeted patient population, generally means that the injured person is paralyzed from the neck down and requires round-the-clock care. The lifetime direct costs of care for a patient suffering a high cervical spinal cord injury, such as a C-4 spinal cord injury, can approach
In addition, the amendment to inclusion parameters will also expand the dosing window to 21 to 42 days after a patient's spinal cord injury occurs, providing study investigators more time to screen patients to determine if they are eligible to participate in the SCiStar study. The expansion of the dosing window is supported by recent preclinical research that indicated AST-OPC1 cells can durably engraft at a patient's injury site when administered up to two months after the date of injury.
About the SCiStar Trial
The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with subacute, C-4 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the
Asterias has received a Strategic Partnerships Award grant from the
AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells originally isolated in 1998, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.
In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. Based on the results of this study, Asterias received clearance from
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