The BioFIND study: Characteristics of a clinically typical Parkinson's disease biomarker cohort.

BACKGROUND: Identifying PD-specific biomarkers in biofluids will greatly aid in diagnosis, monitoring progression, and therapeutic interventions. PD biomarkers have been limited by poor discriminatory power, partly driven by heterogeneity of the disease, variability of collection protocols, and focus on de novo, unmedicated patients. Thus, a platform for biomarker discovery and validation in well-characterized, clinically typical, moderate to advanced PD cohorts is critically needed. METHODS: BioFIND (Fox Investigation for New Discovery of Biomarkers in Parkinson's Disease) is a cross-sectional, multicenter biomarker study that established a repository of clinical data, blood, DNA, RNA, CSF, saliva, and urine samples from 118 moderate to advanced PD and 88 healthy control subjects. Inclusion criteria were designed to maximize diagnostic specificity by selecting participants with clinically typical PD symptoms, and clinical data and biospecimen collection utilized standardized procedures to minimize variability across sites. RESULTS: We present the study methodology and data on the cohort's clinical characteristics. Motor scores and biospecimen samples including plasma are available for practically defined off and on states and thus enable testing the effects of PD medications on biomarkers. Other biospecimens are available from off state PD assessments and from controls. CONCLUSION: Our cohort provides a valuable resource for biomarker discovery and validation in PD. Clinical data and biospecimens, available through The Michael J. Fox Foundation for Parkinson's Research and the National Institute of Neurological Disorders and Stroke, can serve as a platform for discovering biomarkers in clinically typical PD and comparisons across PD's broad and heterogeneous spectrum. © 2016 The Authors. Movement Disorders published by Wiley Periodicals, Inc. on behalf of International Parkinson and Movement Disorder Society.

Therapeutic development paths for cognitive impairment in Parkinson's disease: report of a regulatory roundtable.

Cognitive impairment is a common occurrence in Parkinson's disease (PD), although the severity and specific presentation varies across patients. Initial deficits, including mild cognitive impairment (PD-MCI), may remain stable or in many cases, may progress over variable lengths of time to Parkinson's disease dementia (PDD). As there are currently no marketed treatments for milder forms of cognitive impairment, an opportunity exists to define the path for therapeutic development in this area. In the absence of a well-defined path for the approval of therapies that target PD-MCI, pharmaceutical companies are unlikely to pursue this indication. In order to move forward and improve the quality of life for PD patients, it is imperative for the field to have consensus on the definition of PD-MCI, the best instruments to measure cognitive decline, and a strategy for future clinical trials.