Stem and spine
StemCells’ spinal cord injury Phase 1/2 study yields ‘groundbreaking’ results
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NEWARK, Calif.—The last thing Norwegian cyclist Knut Ølstad remembers before waking up in a hospital bed in 2011 was the elation of flying down the French Alps’ Summet Mount Ventoux. He doesn’t remember the oncoming car or catapulting over the handlebars and crashing onto the road. The diagnosis: complete spinal cord injury. The prognosis: a life of paralysis, dependent on others.
Once a strapping athlete, Ølstad searched for a medical breakthrough, eventually signing on as one of 12 patients participating in StemCells Inc.’s Thoracic Spinal Cord Injury Phase 1/2 study.
“I took a very steep dive from heaven to hell,” Ølstad told DDNews from his home in Oslo. The HuCNS-SC transplantation in the study gave him feeling from his chest to his hips. Ølstad works out two hours a day to strengthen his body while awaiting StemCells’ next breakthrough. “I have high hopes I will walk again,” he says.
StemCells announced the results of the groundbreaking study on May 14, with Dr. Armin Curt, principal investigator, presenting a summary of the safety and preliminary efficacy data from the Phase 1/2 study investigating HuCNS-SC intramedullary transplantation in thoracic spinal cord injury.
“The gains we have detected indicate that areas of sensory function have returned in more than half the patients,” says Curt, who is chairman of the Spinal Cord Injury Center at the University of Zurich’s Balgrist University Hospital. “Such gains are unlikely to have occurred spontaneously given the average time from injury.”
The patient population in the study “represents a form of spinal cord injury that has historically defied responses to experimental therapies, and the measurable gains we have found strongly argue for a biological result of the transplanted cells,” Curt adds. “These gains are exciting evidence that we are on the right track for developing this approach for spinal cord injury. This early outcome in thoracic injury firmly supports testing in cervical spinal cord injury.”
According to Dr. Stephen Huhn, vice president of clinical research and chief medical officer at StemCells, “This research program has the potential to revolutionize the therapeutic paradigm for spinal cord injury patients. The clinical gains observed in this first study are a great beginning. We found evidence of sensory gains in multiple segments of the injured thoracic spinal cord across multiple patients.”
“Our primary focus in this study for spinal cord injury was to evaluate safety and also to look for even small signs of an effect that went beyond the possibility of spontaneous recovery,” Huhn continues. “We are obviously very pleased that the pattern of sensory gains observed in this study are both durable and meaningful, and indicate that the transplantation has impacted the function of damaged neural pathways in the cord.”
Thanks to its early success, StemCells’ development program has now “advanced to a Phase 2 controlled study in cervical spinal cord injury where the corollary of sensory improvements in thoracic spinal cord injury could well be improved motor function in the upper extremities of patients with cervical spinal cord injuries,” he explains.
The international, open-label, Phase 1/2 trial presented by Curt evaluated both safety and preliminary efficacy of StemCells’ proprietary HuCNS-SC human neural stem cells as a treatment for chronic spinal cord injury. The trial enrolled 12 patients (including Ølstad) who had suffered injury to the thoracic cord and were in the early chronic stage of recovery. The severity of spinal cord trauma is classified by the ASIA Impairment Scale (AIS) according to the degree of sensory and motor loss.
Patients classified as AIS A, the most severe injury, have lost both sensory and motor function below the level of injury. AIS B patients are considered less severely injured because, although they also have no motor function below the level of injury, some limited sensory function is maintained.
The protocol was specifically designed to test safety and preliminary efficacy across this spectrum of injury severity, and ultimately the trial transplanted seven AIS A and five AIS B patients.
The patterns of sensory gains were confirmed to involve multiple sensory pathways and were observed more frequently in the patients with less-severe injury; three of the seven AIS A patients and four of the five AIS B patients showed signs of positive sensory gains confirming the previously released interim results. In addition, two patients progressed during the study from the most severe classification, AIS A, to the lesser degree of injury grade, AIS B.
