International Stem Cell Corp. (ISCO-OTCQB) has announced new results from its preclinical Parkinson's disease program. ISCO is developing a novel approach that uses self-renewing human parthenogenetic neural stem cells (hPNSCs), which are derived from the Company’s proprietary histocompatible human pluripotent stem cells, to treat Parkinson’s disease. Parkinson's disease is a progressive disorder of the nervous system that affects four million to six million people worldwide (Source: National Parkinson Foundation). It belongs to a group of motor system disorders that result from the loss of dopamine-producing cells in the brain.
ISCO is currently evaluating its Parkinson's therapy in final preclinical primate studies. Data collected as part of a collaboration with the Sanford Burnham Medical Research Institute, summarized below, was announced at the American Neurological Association's 2013 Annual Meeting in New Orleans.
- The neural stem cells were shown to migrate a significant distance from the site of implantation (in the striatum) to the main region where the loss of dopamine-releasing (“dopaminergic”) neurons occurs in Parkinson's patients—the substantia nigra. No evidence of migration outside the brain was observed.
- Regarding safety, ISCO’s neural stem cells elicited a lower immunogenic response versus other cell types included in the study.
- The study conclusively demonstrated that the neural stem cells were able to differentiate into the specific type of neurons lost in Parkinson’s disease.
ISCO believes that these results establish a solid foundation for the clinical use of neural stem cells for treating Parkinson's disease, and also exposes potential in other neurological indications, such as Alzheimer's disease.
Neural stem cells offer therapeutic benefit through multiple mechanisms of action, including migration to the injury site, secretion of neurotrophic factors (e.g., dopamine), immunomodulation, cell replacement, and recovery of the endogenous neurons. The ability for neural stem cells to replace dopaminergic neurons destroyed by the disease is important in treating Parkinson’s as these cells are responsible for producing and releasing dopamine, which is key to alleviating symptoms of the disease.
Advancing Toward Clinical Trials
The Company is in the process of planning and preparing for first-in-human clinical studies. ISCO has received guidance and feedback on the design of its study from experts in the field of cell therapy and movement disorders. Additionally, in August 2013, the Company contracted Duke University to coordinate its clinical research.
Dependent upon successful submission and approval of an Investigational New Drug (IND) application, the Company anticipates that the Phase I study could commence sometime in 2014.
* * * * * * * * * * *
Visit ISCO’s Corporate Profile for access to
the latest research, news, and stock information
on International Stem Cell Corp.
* * * * * * * * * * *