StemCells, Inc. Receives FDA Authorization of IND for Spinal Cord Injury

Published: October 2, 2013  |  Source: stemcellsinc.com
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stemcellsinc-logoNEWARK, Calif., Oct. 2, 2013 (GLOBE NEWSWIRE) — StemCells, Inc. (Nasdaq:STEM) announced today that the U.S. Food and Drug Administration (FDA) has authorized the Company’s Investigational New Drug (IND) application for clinical testing of the Company’s proprietary HuCNS-SC® human neural stem cells as a treatment for spinal cord injury. As a first action under this IND, the Company is working to open U.S. sites for its Phase I/II clinical trial for chronic spinal cord injury, which is currently underway in Switzerland and Canada.

“This IND is a significant step forward for our spinal cord injury program,” said Stephen Huhn, MD, FACS, FAAP, Vice President, CNS Clinical Research at StemCells, Inc. “With regulatory authorization from Switzerland, Canada and now the United States, we have the first international trial of a stem cell therapy for spinal cord injury. To date, we have transplanted seven patients from Germany, Norway, Italy, Israel, Canada, and the United States at Balgrist University Hospital in Zurich. We now look forward to enrolling patients at U.S. and Canadian sites and easing the travel burden for North American-based patients. Our goal is to complete enrollment in the trial by the first quarter of next year. This is the Company’s fourth FDA-authorized IND, and the agency has now cleared clinical studies of our HuCNS-SC cells in all three elements of the CNS, the brain, the eye and the spinal cord.”

Roman Reed, President of the Roman Reed Foundation, added, “This is great news for the spinal cord community. StemCells has not seen any safety issues in the ongoing trial, and they have reported truly exciting preliminary results, with gains in sensory function persisting for 12 months after transplantation. With this green light from the FDA, it will be much easier for American patients to participate in this innovative trial. This is another step forward in our quest to find a cure for paralysis.”

The Roman Reed Foundation (www.romanreedfoundation.com) is a charitable organization dedicated to the cause of finding cures for neurological disorders, in particular the alleviation of spinal cord injury paralysis.

About the Spinal Cord Injury Clinical Trial

The Company’s Phase I/II clinical trial is designed to assess both safety and preliminary efficacy of HuCNS-SC cells as a treatment for chronic spinal cord injury. The Company plans to enroll twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level, with the injury classified as complete or incomplete according to the American Spinal Injury Association Impairment Scale. To date, seven patients have been enrolled and transplanted with HuCNS-SC cells.

All patients who enroll will receive HuCNS-SC cells through direct transplantation into the spinal cord and will be temporarily immunosuppressed. Patients will be evaluated regularly 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 below the injury site. Preliminary efficacy will be 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 therapy long-term, and each of the patients will be invited to enroll into a separate four-year observational study after completing the Phase I/II study.

Data from the first three patients, each of whom had suffered a complete injury prior to enrolling in the study, showed multi-segment gains in sensory function in two patients twelve months after transplantation of the HuCNS-SC cells. One of the two converted from a complete injury classification to an incomplete injury. The third patient remained stable twelve months after transplantation.

The trial is currently enrolling at Balgrist University Hospital, University of Zurich, a world-leading medical center for spinal cord injury and rehabilitation. Patients who may qualify and are interested in participating in the study should contact the Zurich site either by phone at +41 44 386 3901 or by email at stemcells.pz@balgrist.ch. The Company is working to open trial sites in Canada and the United States.

Information about the clinical trial is available at the ClinicalTrials.gov website of the National Institutes of Health at http://www.clinicaltrials.gov/ct2/show/NCT01321333?lead=StemCells+Inc.&rank=4. 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/Clinical-Trials.htm and at http://www.stemcellsinc.com/Therapeutic-Programs/Spinal-Cord-Injury.htm.

About StemCells, Inc.

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 I 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 conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland, Canada and the United States, and has reported positive interim data for the first three patients. The Company is also conducting a Phase I/II 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 (CIRM). StemCells also markets stem cell research products, including media and reagents, under the SC Proven® brand. Further information about StemCells is available at http://www.stemcellsinc.com.