StemCells receives Swissmedic authorization to initiate HuCNS-SC Phase I/II clinical trial in spinal cord injury

StemCells, Inc. (Nasdaq:STEM) announced today that is has received authorization from Swissmedic, the Swiss regulatory agency for therapeutic products, to initiate a Phase I/II clinical trial in Switzerland of the Company's proprietary HuCNS-SC^® product candidate (purified human neural stem cells) in chronic spinal cord injury. The trial is designed to assess both safety and preliminary efficacy in patients with varying degrees of paralysis who are three to 12 months post-injury, and will progressively enroll patients based upon the severity of injury. Enrollment is expected to begin in early 2011.

The trial will enroll 12 patients with thoracic (chest-level) spinal cord injury, and will include both complete and incomplete injuries as classified by the American Spinal Injury Association (ASIA) Impairment Scale.  The first cohort will include patients classified as ASIA A. These patients have what is considered to be a "complete" injury, or no movement or feeling below the level of the injury. The second cohort will progress to patients classified as ASIA B, or patients with some degree of feeling below the injury. The third cohort will consist of patients classified as ASIA C, or patients with some degree of movement below the injury. In addition to assessing safety, the trial will evaluate preliminary efficacy using defined clinical endpoints, such as changes in sensation, motor, and bowel/bladder function. 

"This trial marks a significant broadening of our clinical development program," said Martin McGlynn, President and CEO of StemCells, Inc. "With this authorization, we will soon have three active clinical trials evaluating our HuCNS-SC cells in conditions affecting both the brain and the spinal cord. Our current clinical investigations now target lysosomal storage disease, myelination disorders and central nervous system trauma."

The trial will be conducted in Zurich, Switzerland at the University Hospital Balgrist, University of Zurich, one of the leading medical centers in the world for spinal cord injury and rehabilitation. The principal investigator is Armin Curt, MD, Professor and Chairman, Spinal Cord Injury Center at the University of Zurich, and Medical Director of the Paraplegic Center at the University Hospital Balgrist. Dr. Curt is an internationally renowned medical expert in spinal cord injury.   

"This trial represents a major advance for the field of spinal cord injury research as effective treatments have yet to be developed for patients living with paralysis," stated Dr. Curt. "The use of neural stem cells to repair an injured spinal cord and restore function is a novel therapeutic approach that I believe holds much promise. Neural stem cells are naturally suited to render repair of the central nervous system, and therefore may prove to be an important strategy for treating spinal cord trauma. I am very excited to be part of a groundbreaking study that could pave the way for developing an effective therapy for this significant unmet medical need." 

The results of numerous preclinical studies, conducted in collaboration with Drs. Aileen Anderson and Brian Cummings at the University of California, Irvine (UCI), demonstrate the significant therapeutic potential of the Company's human neural stem cells for the treatment of spinal cord injury. Data published in several peer-reviewed journals show that the cells engraft, migrate along the spinal cord to the point of injury, and then differentiate into neurons and specialized cells called oligodendrocytes that create the insulation (myelin) necessary for proper transmission of nerve impulses from the brain to below the level of injury. When transplanted in spinal cord-injured mice at both sub-acute and chronic injury time points, the cells have been shown to form protective myelin sheaths around damaged nerve axons and enable a significant and persistent recovery of walking ability. 

Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS program at StemCells, Inc., added, "We feel privileged to be working with such a highly esteemed clinician as Dr. Curt as we seek to develop a therapy with benefits similar to those shown in our preclinical studies. Moreover, this trial design should enhance our ability to evaluate safety and efficacy in a representative population of patients suffering from varying degrees of paralysis." 

Additional information about the Company's spinal cord injury program can be found on the StemCells, Inc. website at http://www.stemcellsinc.com/Therapeutic-Programs/Spinal-Cord-Injury.htm, including video interviews with Company executives and independent collaborators.

More About the Trial

Enrollment will be open to patients in Europe with thoracic spinal cord injury classified as ASIA A, B or C, and with a neurological injury level of T2-T11. All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord, and will be temporarily immunosuppressed. Following transplantation, the patients will be evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery and the immunosuppression, and to measure any recovery of neurological function below the injury site. As the Company intends to follow the effects of this therapy long-term, a separate four-year observational study will be initiated at the conclusion of this trial.