Tuesday, July 1, 2014

International Stem Cell Corporation Completes Important Study in Parkinson’s Disease Program


CARLSBAD, CA - (Marketwired) - 6/19/2014 , International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com) (“ISCO” or “the Company”), a California-based biotechnology company developing novel stem cell based therapies, announced today the completion of the acute toxicity study of the Company’s proposed clinical product to treat Parkinson’s disease.  The recently completed IND-enabling study transplanting rodents with human neural stem cells, derived from ISCO’s proprietary parthenogenetic stem cell platform, showed that the cells are well tolerated even at high doses up to the equivalent of 2.2 billion cells in humans.  The program continues to progress according to the plan outlined with the FDA at the pre-IND meeting as reported in February 2014.  The Company intends to provide a further update early next quarter.
“I’m pleased that we have successfully achieved another important milestone in our Parkinson’s disease program.  A critical part of the development process, and the preparations for the IND submission, was to establish the highest tolerated dose of our clinical product in rodents,” said Dr. Ruslan Semechkin, ISCO’s Chief Scientific Officer.  “The results of this study show that the doses we will be proposing to the FDA in our phase I clinical study design for the treatment of Parkinson’s disease are well below the safety threshold.”

The study consisted of 19 immune deficient rodents divided into four cohorts, a control group and three treatment groups receiving escalating doses of human parthenogenetic neural stem cells (hPNSC).  Animals received stereotactic injections of hPNSC in the striatum and substantia nigra.  The rodents were observed for seven days post transplantation and demonstrated normal appearance and behaviors.  Necropsies showed no abnormal tissues or tumors after sacrifice.  Detailed immunohistochemical analysis demonstrated that the majority of the implanted cells survived the surgery and engrafted into the host brain tissue.

According to the Parkinson’s Disease Foundation, an estimated seven to 10 million people worldwide live with Parkinson’s disease, with as many as one million of those in the United States alone, more than the combined total of people diagnosed with multiple sclerosis, muscular dystrophy, and Lou Gehrig’s disease.  The total direct and indirect cost of Parkinson’s disease is estimated to be nearly $25 billion per year in the United States alone.

ISCO's Parkinson's disease program uses human parthenogenetic neural stem cells (hPNSC), a novel therapeutic cellular product derived from the company's proprietary histocompatible human pluripotent stem cells.  hPNSC are self-renewing multipotent cells that are precursors for the major cells of the central nervous system.  The ability of hPNSC to (i) differentiate into dopaminergic (DA) neurons and (ii) express neurotrophic factors such as glial derived neurotrophic factor (GDNF) and brain derived neurotrophic factor (BDNF) to protect the nigrostriatal system, offers a new opportunity for the treatment of Parkinson's disease, especially in cases where current small molecule approaches fail to adequately control the symptoms.

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, UniStemCellTM. 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.

ISCO's Parkinson's disease program uses human parthenogenetic neural stem cells (hPNSC), a novel therapeutic cellular product derived from the company's proprietary histocompatible human pluripotent stem cells.  hPNSC are self-renewing multipotent cells that are precursors for the major cells of the central nervous system.  The ability of hPNSC to (i) differentiate into dopaminergic (DA) neurons and (ii) express neurotrophic factors such as glial derived neurotrophic factor (GDNF) and brain derived neurotrophic factor (BDNF) to protect the nigrostriatal system, offers a new opportunity for the treatment of Parkinson's disease, especially in cases where current small molecule approaches fail to adequately control the symptoms.
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Contacts:
International Stem Cell Corporation
Dr. Simon Craw
Executive Vice President
Phone: 760-940-6383

Dr. Ruslan Semechkin
Chief Scientific Officer
Phone: 760-940-6383

Email: ras@intlstemcell.com

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