TY - JOUR
T1 - Stereotaxic intrastriatal implantation of human retinal pigment epithelial (hRPE) cells attached to gelatin microcarriers
T2 - A potential new cell therapy for Parkinson's disease
AU - Watts, R. L.
AU - Raiser, C. D.
AU - Stover, N. P.
AU - Cornfeldt, M. L.
AU - Schweikert, A. W.
AU - Allen, R. C.
AU - Subramanian, T.
AU - Doudet, D.
AU - Honey, C. R.
AU - Bakay, R. A.E.
PY - 2003
Y1 - 2003
N2 - 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.
AB - 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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U2 - 10.1007/978-3-7091-0643-3_14
DO - 10.1007/978-3-7091-0643-3_14
M3 - Article
C2 - 12946059
AN - SCOPUS:0141940594
SN - 0303-6995
SP - 215
EP - 227
JO - Journal of Neural Transmission, Supplement
JF - Journal of Neural Transmission, Supplement
IS - 65
ER -