Stereotaxic intrastriatal implantation of human retinal pigment epithelial (hRPE) cells attached to gelatin microcarriers: a potential new cell therapy for Parkinson's disease

J Neural Transm Suppl. 2003:(65):215-27. doi: 10.1007/978-3-7091-0643-3_14.

Abstract

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 (approximately 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.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain Tissue Transplantation*
  • Cell Transplantation*
  • Corpus Striatum / surgery*
  • Epithelial Cells / transplantation
  • Gelatin
  • Humans
  • Parkinson Disease / surgery*
  • Pigment Epithelium of Eye / transplantation*
  • Stereotaxic Techniques*
  • Tomography, Emission-Computed

Substances

  • Gelatin