International Stem Cell Corporation (ISCO), a California-based biotechnology company, has reported positive 12-week results from its pre-clinical in vivo Parkinson's disease study. The primary goal of this behavioural study was to demonstrate the therapeutic benefits of neuronal cells derived from human parthenogenetic stem cell (hpSC) line in a rat model of Parkinson's disease (PD).

Parkinson's disease is a common neurodegenerative disorder caused by a progressive degeneration of dopamine-producing neurons resulting in gradual dysfunction of the motor system and can eventually lead to death. Pharmacological treatments are useful in the relatively early stage of the disease, but the continuous use of pro-dopaminergic medication eventually becomes ineffective and can cause some of the motor symptoms to worsen.

Our proprietary technology is based on a homogeneous population of neuronal cells (NCs) derived from homozygous hpSCs through a scalable and efficient method, developed by ISCO's R&D team. These parthenogenetically-derived NCs are cryopreservable and can become neurons once they are implanted into the brain. As such, they hold significant therapeutic potential not only because they can differentiate into dopamine-producing neurons, but also because these cells deliver trophic factors that may be able to provide a level of protection to existing neurons affected by the disease.

The animal model used in this study is the 6-OHDA lesioned rat, a well-established and validated model of PD which has been used extensively in the development and testing of drugs for the treatment of PD. The pharmacological induction of rotational behavior in rats is widely used to assess the effects of lesions and potential of cell therapy to effectively replace the dopaminergic system in the rat brain and thus serves as a model of PD. The experimental rats with unilateral dopamine (6-OHDA) lesions survived the inoculation of cells into the brain and signs of improvement in rotational behavior of these animals were clearly observed. Correlational analysis of rotation intensity demonstrated a difference between the drug effects in the control group vs. experimental (transplanted) group of animals. These interim results demonstrate that a single injection of hpSC-derived neuronal cells into the striatum of rats with induced PD symptoms can lead to a significant slowdown in the progression of the disease.

Dr Ruslan Semechkin, vice president - head of R&D, said, "This is a very important result for our pre-clinical Parkinson's programme. The initial in vivo results are very encouraging and show the therapeutic promise of hpSC-derived neuronal cells in the treatment of individuals with Parkinson's disease. Results from this behavioural study will be presented and discussed together with the results of non-human primate study before the end of the first quarter of 2013."

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.