International Stem Cell Corporation Announces Positive Animal Efficacy Results in Liver Disease Program
Published: Jan 07, 2013
CARLSBAD, CA--(Marketwire - January 07, 2013) - International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) ("ISCO" or "the Company") a California-based biotechnology company, today announced positive top line efficacy results from its pre-clinical in vivo liver study. The primary goal of the efficacy study was to demonstrate the therapeutic equivalence of human parthenogenetic stem cell (hpSC)-derived hepatocytes to adult liver cells as a prerequisite for using such cells in the treatment of metabolic liver diseases including Crigler-Najjar Syndrome.
Crigler-Najjar Syndrome (CNS) is a rare inherited disorder in which bilirubin, a substance made by the liver, cannot be broken down by the liver. The build-up of this toxic compound can lead to damage to the brain, muscles, and nerves and eventually cause death. Current treatment paradigms for CNS include phototherapy and blood transfusions, but these do not treat the underlying cause of the disease. Hepatocyte transplantation has emerged as a therapeutic strategy, and has been successfully applied to treat patients with CNS, however the extremely limited availability of human livers and therefore of donated primary hepatocytes makes a stem cell based approach attractive.
These ISCO study results demonstrate that the hpSC-derived hepatocytes engraft in the liver of Gunn rats and behave in a similar manner to primary human hepatocytes. The Gunn rat is a well-established model of CNS and has been used extensively to study bilirubin toxicity and hepatocytes transplantation. Additionally, the study indicates that a single intrasplenic injection of hpSC-derived hepatocytes results in a change in the plasma indirect bilirubin level equivalent to that achieved by injecting primary hepatocytes. Establishing the equivalence of hpSC-derived and donor-derived hepatocytes in their ability to metabolize bilirubin supports the thesis that hpSC-derived hepatocytes can be used therapeutically as a substitute for donated primary liver cells.
"Achieving this milestone is very encouraging," said Dr. Andrey Semechkin, CEO and Co-chairman of ISCO. "These results suggest that hpSC-derived hepatocytes could be a well suited alternative to donated primary hepatocytes as a source of cells in clinical applications including the treatment of Crigler-Najjar Syndrome."
About International Stem Cell Corporation
International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.
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Statements pertaining to anticipated developments, the potential use of technologies to develop therapeutic products and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects" or "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products and the management of collaborations, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.
International Stem Cell Corporation
Dr. Simon Craw
Executive Vice President
Dr. Ruslan Semechkin
Vice President, R&D
Senior Vice President