Stereotaxic intrastriatal implantation of human retinal pigment epithelial (hRPE) cells attached to gelatin microcarriers: a potential new cell therapy for Parkinson's disease
Watts, R.L.; Raiser, C.D.; Stover, N.P.; Cornfeldt, M.L.; Schweikert, A.W.; Allen, R.C.; Subramanian, T.; Doudet, D.; Honey, C.R.; Bakay, R.A.E.
Journal of Neural Transmission. Supplementum 2003(65): 215-227
2003
ISSN/ISBN: 0303-6995 PMID: 12946059 Document Number: 552626
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(R)) 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 (apprx325,000 cells) implanted in the most affected post-commissural putamen. All patients tolerated the implantation of Spheramine(R) 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(R) appears to show promise in treating late stage PD patients.