The BioFIND Study (Fox Investigation For New Discovery Of Biomarkers In Parkinson’s Disease): Design And Methodology (P4.043)

OBJECTIVE: To describe the study design of BioFIND, an observational, cross-sectional, multi-center study of biomarkers in moderate to advanced Parkinson’s disease (PD) and healthy control (HC) subjects. BACKGROUND: PD-specific biomarkers will greatly aid clinicians’ ability to diagnose the disease and monitor and/or predict its progression as well as provide a critical tool for therapeutic trials. However, prior studies are largely limited by their sample size, inclusion of heterogeneous PD populations, or focus on de novo, unmedicated PD patients. Thus, a platform for discovery and validation of biomarkers in well-characterized, moderate-to-advanced PD is critically needed. DESIGN/METHODS: BioFIND aims to establish a repository of clinical data, blood, DNA, RNA, and cerebrospinal fluid (CSF) samples from HCs and typical, moderate to advanced PD patients, whose clinical diagnosis is strengthened by a minimum 5 year history and medication response. RESULTS: BioFIND will enroll 120 PD patients and 120 HCs over 2 years. Inclusion criteria for PD include classic motor features, dopaminergic medication benefit, age 55-93 years, PD duration 5-18 years, and PD onset age 50-75 years. Primary exclusion criteria include potential genetic factors (i.e., PD in first-degree relative), atypical parkinsonism or other neurological disorders, or contraindications for lumbar puncture. Evaluations include demographics, motor and non-motor questionnaires, and blood and CSF samples obtained in a practically defined “off medication” state. Presently, 25 PD patients and 6 HCs have been enrolled. Biosamples will be available under the auspices of a program sponsored by The Michael J. Fox Foundation (MJFF) with support from the National Institute of Neurological Disorders and Stroke (NINDS). CONCLUSIONS: The BioFIND study will provide a valuable biospecimen resource for the discovery and validation of novel biomarkers in typical, moderate to advanced PD. Promising markers can then be verified in longitudinal cohorts and more heterogeneous PD subjects. Study Supported by: This study is sponsored by MJFF with support from NINDS. We also thank supports from NIH CTSA (RNA). Disclosure: Dr. Kang has received personal compensation for activities with Caremark Inc. as a medical advisory board member. Dr. Kang has received research support from Allergan, Inc. Dr. Alcalay has received personal compensation for activities with Genzyme. Dr. Alcalay has received research support from the National Institutes of Health, Brookdale Foundation, Parkinson9s Disease Foundation, Smart Foundation, and the Michael J Fox Foundation. Dr. Goldman has received personal compensation for activities with American Academy of Neurology, Movement Disorder Society, Johns Hopkins Dystonia and Spasticity Practicum, and Teva Neuroscience. Dr. Goldman has received research support from NIH/NINDS and the Parkinson9s Disease Foundation. Dr. Henchcliffe has received personal compensation for activities with GE Healthcare, GlaxoSmithKline, Inc., UCB Pharma, Impax, Teva Neuroscience, US World Meds, Gerson Lehman Group, and MedIQ. Dr. Henchcliffe has received personal compensation in an editorial capacity for Neurology Alert. Dr. Henchcliffe has received research support from Kaneka. Dr. Hogarth has nothing to disclose. Dr. Paul Tuite has received research support from General Electric. Dr. Xie has nothing to disclose. Dr. Frasier has nothing to disclose. Dr. Kopil has nothing to disclose. Dr. Vincent has nothing to disclose. Dr. Willis has nothing to disclose. Dr. Casaceli has nothing to disclose. Dr. Rudolph has nothing to disclose.