Summary
Human retinal pigment epithelial (hRPE) cells are dopaminergic support cells in the neural retina. Stereotaxic intrastriatal implantation of hRPE cells attached to gelatin microcarriers (Spheramine®) in rodent and non-human primate models of Parkinson’s disease (PD) produces long term amelioration of motor and behavioral deficits, with histological and PET evidence of cell survival without immunosuppression. Long-term safety in cynomologous monkeys has also been demonstrated.
Six H&Y stage III/IV PD patients were enrolled in a one-year, open-label, single center study to evaluate the safety and efficacy of Spheramine (−325,000 cells) implanted in the most affected post-commissural putamen. All patients tolerated the implantation of Spheramine® well and demonstrated improvement. At 6, 9, and 12 months post-operatively, the mean UPDRS-Motor score “off”, the primary outcome measure, improved 33%, (n = 6), 42% (n = 6), and 48% (n = 3), respectively. No “off-state” dyskinesias have been observed. Based on these preliminary results, Spheramine® appears to show promise in treating late stage PD patients.
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Watts, R.L. et al. (2003). Stereotaxic intrastriatal implantation of human retinal pigment epithelial (hRPE) cells attached to gelatin microcarriers: a potential new cell therapy for Parkinson’s disease. In: Horowski, R., et al. Advances in Research on Neurodegeneration. Journal of Neural Transmission. Supplementa, vol 65. Springer, Vienna. https://doi.org/10.1007/978-3-7091-0643-3_14
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DOI: https://doi.org/10.1007/978-3-7091-0643-3_14
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