The professor of neurosurgery at Rush University Medical School discussed data seen in the phase 1/2 clinical trial of LCTOPC1.
“This is 1 step in a long sequence of research that I have been involved with for more than 30 years. The original research in this area was conducted before we had stem cells and utilized human embryonic spinal cord in patients who had chronic spinal cord injuries in the thoracic spine and who had syringomyelia so that they needed an operation one way or the other.”
Lineage Cell Therapeutics’ oligodendrocyte progenitor cell therapy LCTOPC1 has been well-tolerated in patients with subacute cervical spinal cord injuries, with promising safety and neurological data that indicate further investigation of the therapy.
The phase 1/2 trial (NCT02302157) has dosed 25 patients. All patients experienced at least 1 adverse event (AE) for a total of 534 adverse events. A single case of self-resolving grade 2 dysesthesia was deemed to be potentially related to the LTCOPC1 cells and none of the 29 serious AEs reported were deemed related to the cell therapy.
At 1-year follow-up, 21/22 (96%) of the intention-to-treat group recovered 1 or more levels of neurological function on at least 1 side of their body, and 7/22 (32%) recovered 2 or more levels of neurological function on at least 1 side of their body.
CGTLive spoke with the study’s principal investigator Richard Fessler, MD, professor of neurosurgery, Rush University Medical School, to learn more about the data seen in the trial. He discussed how therapies have evolved throughout his tenure in working with spinal cord injuries.
“Recently published results from our 25-patient phase 1/2a clinical trial support the case for further development of OPC1. This study is one of the longest-running clinical trials in the field of cell therapy, and has provided important information on the safety of the oligodendrocyte cells which we manufacture and deliver to patients. In the earliest-treated patients, with 10 years of follow-up, there were no medical or neurological complications to indicate that the OPC1 cell transplant therapy was unsafe, there were no unexpected serious adverse events attributable to the OPC1 cell implant, and none of the patients treated in that trial had deterioration in neurologic motor function, despite suffering from severe injuries,” Brian Culley, chief executive officer, Lineage Cell Therapeutics, also told CGTLive.
“Lineage has spent significant efforts to develop what we believe will be a superior, second-generation product: a new thaw-and-inject formulation with fewer impurities, more analytical tests to monitor our material, and a commercially feasible production scale. We also have learned much from this first trial about patient selection and assessments, which we believe may lead to better clinical outcomes in later-stage trials which we are planning. Our immediate efforts are focused on demonstrating the safety of a new and improved delivery device, a study which is expected to enroll just 5 to 10 patients, but which will include for the first time patients with chronic spinal cord injuries. Our overall objective is to demonstrate that replacing specific cells of the spinal cord can lead to greater mobility and quality of life for people who have been paralyzed by a severe spinal cord injury,” Culley added.