Long-Term Studies Reveal Positive Results for Initial Phase of Spinal Cord Injury Clinical Trial of Regenerative Medicine-Based Treatment
FDA clears company and research sites, including Shepherd Center, to expand clinical trial to increased numbers of newly injured patients and those with sensory incomplete spinal cord injury.
Asterias Biotherapeutics Inc., a California-based biotechnology company focused on regenerative medicine, recently announced positive results from long-term follow-up studies of five patients – including two treated at Shepherd Center – with thoracic-level spinal cord injury (SCI) who participated in the company’s clinical trial of an investigational product.
Results from Asterias’ Phase 1 clinical trial assessing the safety of a relatively low dose (2 million cells) of AST-OPC1 (oligodendrocyte progenitor cells) administered within 14 days of injury showed, in four of the five patients, reduced spinal cord cavitation as measured by serial MRI scans throughout the four- to five-year follow-up period.. Also, AST-OPC1 appeared to have positive long-term effects on reducing spinal cord tissue deterioration.
“This new long-term follow-up data continues to support the general safety of AST-OPC1 and indicates minimal risk of the transplanted cells having unintended effects,” said Edward Wirth III, M.D., chief medical officer for Asterias. “In detailed immune response monitoring of patients, the results are consistent with long-term cell engraftment, immune system tolerability and an absence of adverse effects. In short, AST-OPC1 does not appear to present any immunological or other long-term safety issues when administered to patients with spinal cord injuries.”
In addition, Asterias announced that recent successes in both the Phase 1 and Phase 1/2a clinical trials of AST-OPC1 supported the FDA’s recent decision to clear expansion of the current Phase 1/2a trial, named SCiStar, from 13 to up to 35 newly injured patients. In the SCiStar trial, the company and its research sites, including Shepherd Center, are testing escalating doses of AST-OPC1 that researchers believe may improve motor function in patients with cervical-level SCI.
The current clinical trial is enrolling patients with a complete loss of sensory and motor function (AIS-A). With the amendment, the FDA also cleared Asterias to extend enrollment to those individuals with partially retained sensory function (AIS B) and has expanded the age range of trial participants to 18 to 69 years.
The goal of the SCiStar trial is to rescue some motor function in patients with cervical SCI so they can become more independent in their activities of daily living, Dr. Wirth said. These patients will be monitored for 15 years to evaluate the safety and success of their treatment.
“Shepherd Center clinicians and researchers are encouraged by these early positive results and hopeful that this expanded clinical trial will result in a treatment that will improve the lives of people with spinal cord injury,” said Donald P. Leslie, M.D., medical director of Shepherd Center and the principal investigator for Shepherd Center for this clinical trial.
Two Shepherd Center patients with cervical spinal cord injuries have participated in the Phase 1/2a clinical trial of AST-OPC1 to date. Researchers and clinicians continue to evaluate newly injured patients to see if they are eligible to participate in the trial.
The first Phase 1/2a patient treated at Shepherd Center in June 2015 progressed from a complete ASIA Impairment Scale (AIS) A injury to an incomplete AIS C injury during the first three months following treatment with a relatively low dose of 2 million cells, intended to test the safety of the injection procedure used to treat these patients. “This progress is very encouraging and is observed in fewer than 5 percent of our AIS A patients at the early stage of their recovery,” Dr. Leslie noted.
The second Phase 1/2a patient was treated at Shepherd Center in April 2016 and received 10 million cells as part of the second cohort of patients enrolled in the Phase 1/2a trial. This patient is now completing rehabilitation.
The open-label, single-arm SCiStar trial is being conducted at six medical facilities, including Shepherd Center, and will include up to 12 centers in the United States. The SCiStar trial is testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells. AST-OPC1 is administered 14 to 30 days post-injury. Patients are being followed by neurological exams and imaging methods to assess the safety and activity of the product.
Additional information on the Phase 1/2a study, including trial sites and eligibility criteria, can be found at www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar study website (www.scistarstudy.com).
All clinical trial participants at Shepherd Center must be patients who are admitted to Shepherd Center. Also, clinical trial eligibility requirements apply. Medical professionals are invited to promptly refer patients for assessment to determine whether they are appropriate for admission to Shepherd Center. Contact Shepherd Center Admissions at 800-SHEPHERD (800-743-7437) or firstname.lastname@example.org.
For more information about Asterias, visit www.asteriasbiotherapeutics.com.
Shepherd Center, located in Atlanta, Georgia, is a private, not-for-profit hospital specializing in medical treatment, research and rehabilitation for people with spinal cord injury, brain injury, multiple sclerosis, spine and chronic pain, and other neuromuscular conditions. Founded in 1975, Shepherd Center is ranked by U.S. News & World Report among the top 10 rehabilitation hospitals in the nation. In its more than four decades, Shepherd Center has grown from a six-bed rehabilitation unit to a world-renowned, 152-bed hospital that treats more than 900 inpatients, 575 day program patients and more than 7,100 outpatients each year.