Abstract Details

Title In Vivo Efficacy Study of Stem Cell Derived Neuronal Cells for the Treatment of Parkinson's Disease

Topic Movement Disorders

Presentation(s) S23 - (-)

Poster/Presentation
Number 007

Objective

Background Human parthenogenetic stem cells (hpSCs) are pluripotent stem cells that have an important clinical advantage when used in cell replacement therapy because of their homozygosity at the HLA loci, which simplifies immune matching with potential significantly reduced immunogenicity. As such, hpSC are useful for generation of dopaminergic neurons for Parkinson's disease cell therapy.

Design/Methods Highly pure populations of neural stem cells (NSCs) and dopaminergic (DA) neurons were generated from hpSCs and transplanted into rodent and non-human primate models of Parkinson's disease. In the rodent model, behavioral improvement is assessed in 6-OHDA rats with the cylinder test, amphetamine and apomorphine induced rotation tests. In the non-human primate model, behavioral improvement is assessed in MPTP-induced African green monkeys with parkinsonian scores. The intake of food and water is monitored on a daily basis and body weight is recorded weekly. Cell engraftment, viability and phenotype of the implanted cells are determined through histology and HPLC at the end of the studies. Tumorigenicity and safety of the therapy is assessed at the end of both studies by gross necropsy.

Results Preliminary results of the animal studies have shown that the neuronal cells engraft in a Parkinsonian rat model, release dopamine in vivo, and do not give rise to tumors in the brain. We are currently evaluating the post-implantation behavioral improvement of transplanted NSCs and DA neurons precursors in rodent and non-human primate models of Parkinson's disease.

Conclusions The results of our pre-clinical animal studies indicate that neuronal cells derived from human parthenogenetic stem cell can be a viable alternative for the treatment of Parkinson's disease.

Authors/Disclosures
Russell A. Kern, PhD (International Stem Cell Corporation) PRESENTER	No disclosure on file
Ibon Garitaonandia (International Stem Cell Corporation)	No disclosure on file
Rodolfo Gonzalez	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Rosario Sanchez-Pernaute	No disclosure on file
Evan Y. Snyder, MD, PhD (The Burn Institute)	No disclosure on file
Guy J. Buckle, MD (Biogen)	No disclosure on file

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