Time to change the blind men and the elephant approach to Parkinson disease?

Abstract

Time to change the blind men and the elephant approach to Parkinson disease? ABSTRACTParkinson disease (PD) is a progressive neurodegenerative disease that is associated with substantial morbidity and early mortality. Disease-related costs exceed $10 billion, not including medications, out-of-pocket expenses, or societal costs. Symptomatic treatment with levodopa, which has been available for over 30 years, and advanced therapies such as deep brain stimulation improve outcomes. Yet most new medications for PD provide a therapeutic benefit that is relatively modest compared to the benefits from levodopa. Despite dozens of neuroprotective clinical trials, there are no medications proven to slow the progression of the disease. Given these limitations, we provide evidence of the potential public health impact of a research agenda that emphasizes identification of risk factors to reduce disease burden through exposure mitigation. In addition, we emphasize health care policy that focuses on increasing health care expenditures for neurologic evaluation and management services to increase access to specialists to improve disease outcomes and reduce costs through better disease management. Neurology ® 2015;85:190-196 GLOSSARY AOR 5 adjusted odds ratio; CI 5 confidence interval; CMS 5 Centers for Medicare and Medicaid Services; E&M 5 evaluation and management; OR 5 odds ratio; PAR 5 population attributable risk; PD 5 Parkinson disease; tPA 5 tissue plasminogen activator; UPDRS 5 Unified Parkinson's Disease Rating Scale.In the story "Elephant and the blind men," 1 6 blind men discover a creature (an elephant) in their village that they have never encountered. They touch the various parts of the elephant and describe the elephant as a pillar, rope, tree trunk, wall, and tree branch. They argue bitterly until a wise man tells them all of their analyses are accurate, but incomplete. A common interpretation of that story is that without the big picture, one can easily come to a false conclusion. In many ways, the current research approach to Parkinson disease (PD) is similar to the "Elephant and the blind men." We have made substantial advances in understanding PD, but the big picture eludes us. In the following paragraphs, we argue that we have the potential to reduce PD burden and morbidity considerably by shifting to the clinical and research approach used in stroke, namely a public health approach that focuses providing access to the most effective treatments without disparity and prioritizing primary prevention and health outcomes research.The National Institutes for Neurologic Disease and Stroke recently announced the failure of the .$60 million NIH Exploratory Trials in Parkinson's Disease trial of creatine to demonstrate efficacy in slowing PD progression. This joins a long list of failed disease-modifying PD trials, including selegiline, 2,3 riluzole, CEP-1347, 4 rasagiline, 5 creatine, 6 and cell-based therapies. 7,8 Possible explanations for these disappointments include disease models that do not replicate neuro...

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