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Neurology supplements are not peer-reviewed. Information contained in Neurology supplements represent the opinions of the authors and are not endorsed by nor do they reflect the views of the American Academy of Neurology, Editor-in-Chief, or Associate Editors of Neurology.

Gene transfer of trophic factors and stem cell grafting as treatments for Parkinson’s disease

From the Department of Neurological Sciences, Rush University Medical Center, Chicago, Illinois (Drs. Dass and Kordower), and the Departments of Neurology and Neuroscience, Mount Sinai School of Medicine, New York, New York (Dr. Olanow).

Address correspondence and reprint requests to Dr. Jeffrey H. Kordower, Department of Neurological Sciences, 1735 W. Harrison St., Rush University Medical Center, Chicago, IL 60657; e-mail: jkordowe{at}rush.edu

Current therapies for Parkinson’s disease (PD) are limited in their ability to control PD symptomatology, are associated with motor and psychiatric side effects, and do not prevent disease progression. Considerable scientific and media interest has focused on the potential value of gene and stem cell therapies to overcome these problems and to enhance the quality of life for PD patients. Gene therapies utilize a viral vector to deliver a protein of interest to specific brain region. Clinical trials of gene therapy are currently underway using adeno-associated virus to deliver AADC to the striatum, the trophic factor nurturin to the striatum, and GAD to the STN. To date, no serious adverse effects have been noted, but only a small number of patients have been studied. Stem cells are pluripotential cells that offer the potential of generating unlimited numbers of optimized dopamine cells for transplantation. Stem cells can be grown and expanded in tissue culture and then induced to differentiate into dopamine neuronal phenotypes. Transplantation of these cells into the striatum is associated with behavioral improvement in 6-OHDA rodents and MPTP monkeys. Still, only small numbers of transplanted dopaminergic cells survive, and benefits are modest. Clinical trials in PD have not yet been performed. There is considerable enthusiasm for the potential of these procedures, but there remains much to learn in the laboratory and neither has been established to be effective as a treatment for PD. Long term safety and efficacy trials have not been performed in PD patients and the potential of unanticipated side effects must be addressed. Further, neither treatment is expected to improve the non-dopaminergic features of PD.

Publication of this supplement was supported by an educational grant from Teva Neuroscience and Eisai, Inc.

Disclosure: The sponsor has provided CWO with personal honoraria (in excess of $10,000) during his professional career. The sponsor has provided JHK with an honorarium for his participation in this project and personal honoraria during his professional career. BD reports no conflict of interest.

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