Objective: Establish an Industry-Academic collaboration to identify factors contributing to disease progression in PD.

Background: Ashkenazi Jews (AJ) are an excellent ethnic group in which to study prodromal Parkinson’s disease (PD), due to the high frequency of the LRRK2-G2019S mutation (14% of AJ PD patients) and 7 GBA mutations (19% of AJ PD patients). Biogen and the Tel-Aviv Medical Center (TASMC) have established a collaborative effort to better characterize clinical and biological attributes underlying disease progression in AJ PD patients, including LRRK2 and GBA mutation carriers.

Method: BeaT-PD began in December 2016, aiming to recruit 800 subjects: 400 PD patients with less than 7 years of motor symptoms, (150 PD LRRK2-G2019S carriers, 150 PD GBA mutation carriers and 100 PD patients with no known mutations) and 400 undiagnosed healthy subjects over the age 40 (150 relatives of AJ PD- LRRK2-G2019S carriers, 150 relatives of AJ PD-GBA carriers, and 100 healthy controls with no family history of PD). Subjects are genotyped, blood and urine are collected and DNA, RNA and epigenetic analysis, and a comprehensive clinical assessment, including in-clinic and at-home wearable biosensors is conducted. All aspects of study planning and analysis have been a joint effort of TASMC and Biogen. 665 subjects are seen once (cross-sectional cohort). A subgroup of 135 subjects is assessed longitudinally every year for 3 years; 45 patients with PD, 60 at-risk relatives and 30 healthy controls. This group will undergo additional assessments including structural and functional brain MRI, DaT, and lumbar puncture for cerebrospinal fluid (CSF).

Results: As of February, 2019, 571 subjects have been recruited: 217 with PD (43 PD-LRRK2-G2019S, 66 PD-GBA, 108 iPD (mean age: 62.3±10.0, 65%Males, disease duration: 2.9±2.3, Hoehn and Yahr:1.7±0.6); 304 patient relatives (mean age: 53.2±10.4, 52% Males) and 50 healthy controls (mean age: 54.6±12.7, 36%Males). 123 subjects completed the first longitudinal assessment; 45 with PD (mean age: 59.1±12.0, 60% Males, disease duration: 2.2±1.9, Hoehn and Yahr: 1.3±0.2), 56 healthy mutation carriers (mean age: 53.1±7.9, 50%Males) and 22 healthy controls (mean age: 55.4±6.3, 50% Males).

Conclusion: This study demonstrates the potential of collaboration between industry and academic medical centers with the hope of advancing a cure for PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/beat-pd-an-industry-academic-collaboration-to-understand-pd-progression-in-ashkenazi-jews/