Human Clinical Data Encompasses Five Years of Post-Transplant Safety
NEWARK, Calif., Oct. 21, 2013 (GLOBE NEWSWIRE) -- StemCells, Inc. (Nasdaq:STEM) announced today the results of a four-year observation study in patients with neuronal ceroid lipofuscinosis (NCL), also referred to as Batten disease, who had been transplanted with the Company's proprietary HuCNS-SC® cells (purified human neural stem cells) in the initial Phase I study. Key results include long-term evidence of safety, up to five years post transplantation, for the surgical transplantation of the HuCNS-SC cells into multiple sites in the brain and at doses of up to one billion cells. The study results represent the first, and thus far only, multi-year data set following transplantation of neural stem cells into human subjects, and supports the feasibility of the Company's approach in multiple neurological disorders. The data will be presented today by Nathan Selden, MD, PhD, FACS, FAAP, who was co-principal investigator in the studies, at the Congress of Neurological Surgeons Annual Meeting in San Francisco, California.
"The NCL study enrolled patients suffering from a severe progressive neurological disorder and the study's outcome shows that there were no long-term safety or tolerability issues associated with the cells, the immunosuppression regimen or the surgical procedure over the five years following transplantation," said Dr. Selden, Campagna Chair of Pediatric Neurosurgery at Doernbecher Children's Hospital and Oregon Health & Science University (OHSU), and incoming President of the Congress of Neurological Surgeons. "Our assessment of the patients' cognitive and neurological function revealed stable scores in some tests, but the clinical outcomes were generally consistent with the expected course of impairment associated with this neurodegenerative disease. However, three of the six patients transplanted with HuCNS-SC cells have now survived more than five years post-transplant, and in a progressive neurodegenerative disorder, it is noteworthy that each have stable quality-of-life measures."
Stephen Huhn, MD, FACS, FAAP, Vice President, CNS Clinical Research at StemCells, Inc., added, "This was the first clinical test of our HuCNS-SC cells. We are very pleased to have accrued significant multi-year human safety and tolerability data for neural stem cell transplantation into the brain, unmatched in terms of both cell dose and duration of follow up thus far in the field. We are very grateful to all the investigators at OHSU and the families of the patients who were part of this ground-breaking study."
Six patients were enrolled in the Company's Phase I clinical study in Batten disease. All six were transplanted with HuCNS-SC cells and followed for twelve months after transplantation. Five patients completed the Phase I study and subsequently enrolled in a four-year, long-term observational study, with three of the five surviving to the end of the four-year study. The long-term clinical data appear to be consistent with the natural history of the disease and conclusions about impacting the disease course cannot be made in an open-label trial. The reported adverse events are consistent with the underlying disease and there have been no safety concerns attributed to the HuCNS-SC cells. Magnetic resonance imaging scans of the brain show progressive atrophy consistent with the patient's neuropsychological performance. Quality-of-life measures remained stable across all three surviving patients.
The Company previously reported post-mortem evidence of engraftment, migration and long-term survival of the HuCNS-SC cells following transplantation and the planned cessation of immunosuppression. The data were based on examination of the brains from three patients who expired from causes related to the underlying disease.
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 also conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and Canada 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) at two trial sites in the US. In addition, the Company is pursuing preclinical studies in Alzheimer's disease, with funding 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.
Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the future business operations of StemCells, Inc. (the "Company"); the prospect for continued clinical development of the Company's HuCNS-SC cells in central nervous system (CNS) disorders; and the timing and nature of data from the Company's clinical studies in NCL. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including uncertainties with respect to the fact that additional trials will be required to confirm the safety and demonstrate the efficacy of the Company's HuCNS-SC cells for the treatment of spinal cord injury, PMD or any other disease or disorder; uncertainties about whether preliminary data in any Phase I clinical study will prove to be reproducible or biologically meaningful; risks whether the FDA or other applicable regulatory agencies will permit the Company to continue clinical testing of its HuCNS-SC cells; uncertainties about the design of future clinical trials and whether the Company will receive the necessary support of a clinical trial site and its institutional review board to pursue future clinical trials in spinal cord injury, PMD, AMD, or in proposed therapies for other diseases or conditions; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current and planned research and development operations; uncertainty as to whether HuCNS-SC cells and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; uncertainties regarding whether data generated in clinical studies of one disease or condition will be predictive of outcomes in other diseases or conditions; uncertainties regarding the Company's manufacturing capabilities given its increasing preclinical and clinical commitments; uncertainties as to whether the Company will become profitable; and other factors that are described under the heading "Risk Factors" disclosed in Part I, Item 1A in the Company's Annual Report on Form 10-K for the year ended December 31, 2012 and in its subsequent reports on Form 10-Q and Form 8-K.
CONTACT: Rodney Young
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