Significant sensory gains after stem cell transplantation

StemCells, Inc. provides update on its Phase 1/2 study in spinal cord injury

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NEWARK, Calif.—StemCells’ principal investigator Armin Curt, M.D., has presented an interim update on the Phase 1/2 trial in spinal cord injury at the Annual Meeting of the American Spinal Injury Association in San Antonio, Texas. Interim analysis of clinical data to date has shown that the significant post-transplant gains in sensory function first reported in two patients have now been observed in two additional patients.

The presentation included the first data on AIS B subjects to be transplanted in the Phase 1/2 chronic spinal cord injury trial with the HuCNS-SC® cells. In contrast to AIS A patients who have no mobility or sensory perception below the point of injury, AIS B subjects are less severely injured; they are paralyzed but retain sensory perception below the point of injury. Two of the three AIS B patients had significant gains in sensory perception and the third remained stable. The interim results also continue to confirm the favorable safety profile of the cells and the surgical implant procedure.

The presentation included data from a total of five new subjects with a minimum six month follow-up. In total, the company has now reported clinical updates on a total of eight of the twelve patients enrolled in its Phase 1/2 clinical trial using the company's proprietary HuCNS-SC (purified human neural stem cells) platform technology for chronic thoracic spinal cord injury.

"Thoracic spinal cord injury was chosen as the indication in this first trial primarily to demonstrate safety. This patient population represents a form of spinal cord injury that has historically defied responses to experimental therapies and is associated with a very high hurdle to demonstrate any measurable clinical change. Because of the severity associated with thoracic injury, gains in multiple sensory modalities and segments are unexpected, and changes in motor function are even more unlikely," said Curt, professor and chairman of the Spinal Cord Injury Center at Balgrist University Hospital, University of Zurich. "In contrast, the cervical cord, which controls more motor function, may represent a patient population in which motor responses to transplant may be more readily anticipated."

"We are seeing multi-segmental gains and a return of function in the cord in multiple patients. This indicates something that was not working in the spinal cord, now appears to be working following transplantation. This is even more significant because of the time that has elapsed from the date of injury, which ranges from 4 months to 24 months across the subjects with sensory gains," said Stephen Huhn, M.D., FACS, FAAP, vice president, CNS clinical research at StemCells, Inc. "These results are exciting with respect to the expansion of this trial into patients with cervical injury because even a gain of one to two segments in cervical spinal cord injury patients can allow for additional function in the upper extremities."

The company's Phase 1/2 clinical trial is designed to assess the tolerability, safety and preliminary efficacy of HuCNS-SC cells as a treatment for chronic spinal cord injury. The company enrolled 12 subjects with thoracic (chest-level) neurological injuries at the T2-T11 level, classified as complete (AIS A) or incomplete (AIS B) according to the American Spinal Injury Association Impairment Scale. Seven of the patients enrolled in the study were classified as AIS A and 5 classified as AIS B.

All twelve patients have been enrolled and transplanted with HuCNS-SC cells. Each of the first three subjects suffered a complete injury prior to enrolling in the study. Twelve months after transplantation of the HuCNS-SC cells, data showed multi-segment gains in sensory function in two of the first three subjects, one of which converted from a complete injury classification to an incomplete injury. The third subject in this cohort remained stable 12 months after transplantation.

The trial enrolled spinal cord injury patients at three centers: the University of Calgary; the University of Toronto; and at Balgrist University Hospital, University of Zurich, all world-leading medical centers for spinal cord injury and rehabilitation.

All subjects who enrolled in the trial received HuCNS-SC cells through direct transplantation into the spinal cord and underwent temporary treatment with immunosuppressive drugs. Evaluations are being regularly performed in the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any change in neurological function. Preliminary efficacy is being evaluated based on defined clinical endpoints, such as changes in sensation, motor function and bowel/bladder function. The company intends to follow the effects of this intervention long term, and each of the subjects will be invited to enroll in a separate four-year observational study after completing the Phase 1/2 study. In addition, the company plans to initiate a controlled Phase 2 efficacy trial in cervical spinal cord injury in 2014.

StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The company's lead therapeutic product candidate, HuCNS-SC cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. In a Phase 1 clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the company has shown preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The company is also conducting a Phase 1/2 clinical trial in dry age-related macular degeneration (AMD) in the United States. In addition, the company is pursuing preclinical studies in Alzheimer's disease, with support from the California Institute for Regenerative Medicine.
Source: Stem Cells, Inc.

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