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Help Cure Parkinson ’ s Disease : Please Don ’ t Waste the Golden Year
By Robert A . Hauser , MD , Professor of Neurology and Director of the Parkinson ’ s Disease and Movement Disorders Center of the University of South Florida
This isn ’ t a solicitation for a financial donation . But I am going to ask for something very valuable . It is in short supply and critical to our getting an approved therapy to slow progression of Parkinson ’ s disease ( PD ), and ultimately to reaching our shared goal of getting to a cure .
The discovery that aggregation of misfolded alpha-synuclein and propagation of this process through the autonomic and central nervous system lies at the heart of PD suggested many targets potentially amenable to therapeutic interventions . Enthusiasm is rightly running high that interruption of this process , or enhancement of mechanisms that clear toxic aggregates will slow or stop disease progression .
However , we still do not have validated biomarkers , such as a blood test , that allow us to assess disease progression and test these promising therapies in people with PD . Although several different clinical trial designs
have been employed , the most common and straightforward methodology to test a promising disease modifying therapy is to assess its ability to slow progression of clinical signs and symptoms versus placebo over time in patients with early PD while they are not receiving symptomatic PD medications ( e . g ., levodopa , dopamine agonists , MAO-B inhibitors ).
But we have a fundamental challenge ! Individuals who are diagnosed with early PD based on some combination of bradykinesia ( slow and small movement ), rigidity ( stiffness ), and tremor ( shaking ), can only be followed without institution of symptomatic medication for about 6 – 12 months . After that time , a substantial proportion of patients will want or need symptomatic medication to relieve increasing signs and symptoms .
Ideally , we would like to be able to diagnose motor PD as early as possible and follow