Intrastriatal implantation of human retinal pigment epithelial cells attached to microcarriers in advanced Parkinson disease

Natividad P. Stover, Roy A.E. Bakay, Thyagarajan Subramanian, Cathy D. Raiser, Michael L. Cornfeldt, Alfred W. Schweikert, Richard C. Allen, Ray L. Watts

Research output: Contribution to journalArticle

105 Citations (Scopus)

Abstract

Background: Human retinal pigment epithelial (RPE) cells produce levodopa and can be isolated from postmortem human eye tissue, grown in culture, and implanted into the brain attached to microcarriers. These implants ameliorated the motor deficits in rodent and nonhuman primate models of Parkinson disease. Objective: To evaluate the safety and efficacy of unilateral implantation of human RPE cells attached to gelatin microcarriers into the putamen contralateral to the more symptomatic side of patients with Parkinson disease. Design: Open-label pilot study. Setting: A tertiary referral center for movement disorders. Patients: Six patients with advanced Parkinson disease. Interventions: We performed stereotactic intrastriatal implantation of approximately 325 000 RPE cells on microcarriers. Main Outcome Measure: Change from baseline to 12 months in the Unified Parkinson's Disease Rating Scale motor subscore with the patients in the practically defined off state (not taking antiparkinsonian medications for at least 12 hours overnight). Results: The implants were well tolerated. We observed an average improvement of 48% at 12 months after implantation in the Unified Parkinson's Disease Rating Scale motor subscore with the patient in the off state, which was sustained through 24 months. Improvement was also observed in activities of daily living, quality of life, and motor fluctuations. No off-state dyskinesias were observed. Conclusions: Implants of human RPE cells attached to gelatin microcarriers appear to be safe and well tolerated, and they improved motor symptoms in patients with Parkinson disease. On the basis of these results, a randomized, double-blind, placebo-controlled study has been initiated.

Original languageEnglish (US)
Pages (from-to)1833-1837
Number of pages5
JournalArchives of Neurology
Volume62
Issue number12
DOIs
StatePublished - Dec 1 2005

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Retinal Pigments
Parkinson Disease
Epithelial Cells
Gelatin
Antiparkinson Agents
Putamen
Dyskinesias
Movement Disorders
Levodopa
Activities of Daily Living
Tertiary Care Centers
Primates
Cells
Implantation
Pigments
Parkinson's Disease
Rodentia
Placebos
Quality of Life
Outcome Assessment (Health Care)

All Science Journal Classification (ASJC) codes

  • Arts and Humanities (miscellaneous)
  • Clinical Neurology

Cite this

Stover, Natividad P. ; Bakay, Roy A.E. ; Subramanian, Thyagarajan ; Raiser, Cathy D. ; Cornfeldt, Michael L. ; Schweikert, Alfred W. ; Allen, Richard C. ; Watts, Ray L. / Intrastriatal implantation of human retinal pigment epithelial cells attached to microcarriers in advanced Parkinson disease. In: Archives of Neurology. 2005 ; Vol. 62, No. 12. pp. 1833-1837.
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Intrastriatal implantation of human retinal pigment epithelial cells attached to microcarriers in advanced Parkinson disease. / Stover, Natividad P.; Bakay, Roy A.E.; Subramanian, Thyagarajan; Raiser, Cathy D.; Cornfeldt, Michael L.; Schweikert, Alfred W.; Allen, Richard C.; Watts, Ray L.

In: Archives of Neurology, Vol. 62, No. 12, 01.12.2005, p. 1833-1837.

Research output: Contribution to journalArticle

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T1 - Intrastriatal implantation of human retinal pigment epithelial cells attached to microcarriers in advanced Parkinson disease

AU - Stover, Natividad P.

AU - Bakay, Roy A.E.

AU - Subramanian, Thyagarajan

AU - Raiser, Cathy D.

AU - Cornfeldt, Michael L.

AU - Schweikert, Alfred W.

AU - Allen, Richard C.

AU - Watts, Ray L.

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N2 - Background: Human retinal pigment epithelial (RPE) cells produce levodopa and can be isolated from postmortem human eye tissue, grown in culture, and implanted into the brain attached to microcarriers. These implants ameliorated the motor deficits in rodent and nonhuman primate models of Parkinson disease. Objective: To evaluate the safety and efficacy of unilateral implantation of human RPE cells attached to gelatin microcarriers into the putamen contralateral to the more symptomatic side of patients with Parkinson disease. Design: Open-label pilot study. Setting: A tertiary referral center for movement disorders. Patients: Six patients with advanced Parkinson disease. Interventions: We performed stereotactic intrastriatal implantation of approximately 325 000 RPE cells on microcarriers. Main Outcome Measure: Change from baseline to 12 months in the Unified Parkinson's Disease Rating Scale motor subscore with the patients in the practically defined off state (not taking antiparkinsonian medications for at least 12 hours overnight). Results: The implants were well tolerated. We observed an average improvement of 48% at 12 months after implantation in the Unified Parkinson's Disease Rating Scale motor subscore with the patient in the off state, which was sustained through 24 months. Improvement was also observed in activities of daily living, quality of life, and motor fluctuations. No off-state dyskinesias were observed. Conclusions: Implants of human RPE cells attached to gelatin microcarriers appear to be safe and well tolerated, and they improved motor symptoms in patients with Parkinson disease. On the basis of these results, a randomized, double-blind, placebo-controlled study has been initiated.

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