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Future of cell and gene therapies for Parkinson's disease


  • Ole Isacson MD, PhD,

    Corresponding author
    1. Department of Neurology (Neuroscience), Center for Neuroregeneration Research and National Institute of Neurological Disorders and Stroke Udall Parkinson's Disease Research Center of Excellence, Harvard Medical School/McLean Hospital, Belmont, MA
    • Neuroregeneration Laboratories, McLean Hospital, 115 Mill Street, Belmont, MA 02478
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  • Jeffrey H. Kordower PhD

    1. Department of Neurological Sciences, Research Center for Brain Repair, Rush Presbyterian Medical Center, Chicago, IL
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  • Potential conflicts of interest: This article is part of a supplement sponsored by Boehringer Ingelheim (BI). O.I. has acted as a consultant and scientific advisory board member for BI, and has no financial ties, funding, or equity with BI. O.I. has no financial interests in the material presented in this article. J.H.K. has served as a consultant for BI, Ceregene, Genegraft, Nupathe, and Bayer. J.H.K. has no other financial ties, funding, or equity with the sponsor.


The experimental field of restorative neurology continues to advance with implantation of cells or transfer of genes to treat patients with neurological disease. Both strategies have generated a consensus that demonstrates their capacity for structural and molecular brain modification in the adult brain. However, both approaches have yet to successfully address the complexities to make such novel therapeutic modalities work in the clinic. Prior experimental cell transplantation to patients with PD utilized dissected pieces of fetal midbrain tissue, containing mixtures of cells and neuronal types, as donor cells. Stem cell and progenitor cell biology provide new opportunities for selection and development of large batches of specific therapeutic cells. This may allow for cell composition analysis and dosing to optimize the benefit to an individual patient. The biotechnology used for cell and gene therapy for treatment of neurological disease may eventually be as advanced as today's pharmaceutical drug-related design processes. Current gene therapy phase 1 safety trials for PD include the delivery of a growth factor (neurturin via the glial cell line–derived neurotrophic factor receptor) and a transmitter enzyme (glutamic acid decarboxylase and aromatic acid decarboxylase). Many new insights from cell biological and molecular studies provide opportunities to selectively express or suppress factors relevant to neuroprotection and improved function of neurons involved in PD. Future gene and cell therapies are likely to coexist with classic pharmacological therapies because their use can be tailored to individual patients' underlying disease process and need for neuroprotective or restorative interventions. Ann Neurol 2008;64 (suppl):S122–S138