International Stem Cell Corporation Announces Positive Parkinson's Disease Data

Tuesday April 29, 2014

Wall Street Journal Online - International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com), a California-based biotechnology company developing novel stem cell-based therapies and biomedical products announced today that some behavioral improvements have been observed after six months in the pre-clinical non-human primate (NHP) study of Parkinson's disease (PD). The detailed behavioral data will be presented at the 66th American Academy of Neurology Annual Meeting in Philadelphia.

"It is encouraging to see these behavioral scores trending in the right direction as it means that the implanted cells may be having a positive impact on the disease symptoms," said Professor D. Eugene Redmond Jr. MD, of Yale University Medical School and the study's supervisor. "The rating scores are equivalent to components of the UPDRS which is widely used in research to evaluate Parkinson's patients. The Parkison's score is known to correlate very highly with brain dopamine concentrations."

The study consists of 18 primates, all exposed to the neurotoxin, MPTP, divided into three cohorts, a sham treated group and two treatment groups receiving different doses of human neural stem cells (hPNSC) derived from ISCO's proprietary parthenogenetic stem cell line. All of the groups had matching levels of parkinsonism and functional disability prior to the cell injections. The 6 months data showed that the healthy behavior scores of the treatment group increased 170% while that of the placebo group increased by 58%. In addition, one of the treatment groups demonstrated a significant improvement in the main Parkinson's rating score of 63% (p < 0.05) while there was no significant improvement in the control group. The changes in these scores are particularly noteworthy as it signifies a greater reduction in the severity of the symptoms in the treatment group compared with the control group. A more detailed update will be presented once the histopathology and biodistribution analysis of the tissue has been completed.

Dr. Ruslan Semechkin ISCO's Chief Scientific Officer commented: "The results of this interim analysis are very promising. This study provides information about how our human neural stem cells, derived from our parthenogenetic stem cells, behave in a diseased brain and how the diseased tissue responds and is a critical part of our planned IND submission."

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(TM). 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.

According to the Parkinson's Disease Foundation, an estimated seven to 10 million people worldwide live with PD, 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 distrophy, 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.

(29 April 2014). Wall Street Journal Online. International Stem Cell Corporation Announces Positive Parkinson's Disease Datahttp://online.wsj.com/article/PR-CO-20140429-909787.html