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RPE cell implants show promise as Parkinson’s disease therapy
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Implanting retinal pigment epithelial cells into the brain may be a beneficial treatment for Parkinson’s disease, a pilot trial in humans suggests.
Natividad P. Stover, MD, and colleagues treated six patients with advanced Parkinson’s disease with unilateral implantation of human retinal pigment epithelial (RPE) cells attached to gelatin microcarriers. The implants appear to be safe, and they improved motor symptoms in patients with Parkinson’s disease, the study authors reported.
Cultured RPE cells had previously been shown to produce levodopa and to improve motor deficits in animal models of Parkinson’s. Dr. Stover and coworkers noted that in previous studies, human RPE cells have been extracted from postmortem eyes, grown in culture, attached to gelatin microcarriers and implanted into the brain in animals. They performed this study to evaluate the procedure’s safety in humans.
The median age of the patients at baseline was 52.2 years, and they had been diagnosed with Parkinson’s for an average of 10.2 years. Their mean baseline score on a standard Parkinson’s disease rating scale was 118 without medication; on that scale 0 represents no disability and 199 represents a worst-case scenario.
Main outcome measures in the study included the change from baseline to 1 year in the disease scale motor subscore with the patients in the “practically defined off state (not taking medication for at least 12 hours overnight),” according to the study.
The implants were well tolerated, with an average improvement of 48% on the disease scale motor subscore at 1 year after implantation. The improvement was sustained through 2 years, according to the researchers. Improvement was also observed in activities of daily living, quality of life and motor fluctuations. No off-state dyskinesias were observed.
On the basis of these promising results, the study authors said, a randomized, double-masked, placebo-controlled study has begun.
The study is published in the December issue of Archives of Neurology.