Author Affiliations: University Department of Clinical Neurosciences, Royal Free and University College Medical School, and Institute of Neurology, Queen Square, London, England (Dr Schapira); Department of Neurology, Mount Sinai School of Medicine, New York, NY (Dr Olanow).
Parkinson disease is an age-related neurodegenerative disease that affects
approximately 1 million persons in the United States. Current therapies provide
effective control of symptoms, particularly in the early stages of the disease,
but most patients develop motor complications with long-term treatment, and
features develop such as postural instability, falling, and dementia that
are not adequately controlled with existing medications. Accordingly, neuroprotective
therapy that might slow, stop, or reverse disease progression is urgently
needed. While many agents appear to be promising based on laboratory studies,
selecting clinical end points for clinical trials that are not confounded
by symptomatic effects of the study intervention has been difficult. More
recently, neuroimaging end points have been used as biomarkers of disease
progression, but again there are concerns that they may be influenced by regulatory
effects of the drugs used. We review clinical trials aimed at detecting neuroprotection
in Parkinson disease and address the controversies surrounding the interpretation
of these studies.
Schapira AHV, Olanow CW. Neuroprotection in Parkinson Disease: Mysteries, Myths, and Misconceptions. JAMA. 2004;291(3):358–364. doi:10.1001/jama.291.3.358
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