Objective: To identify PSP/CBD progression biomarkers both to improve understanding of disease etiology and course, and to provide crucial tools to enhance the likelihood of success of the disease-modifying therapeutic trials, we have launched a longitudinal observational study for PSP and CBD. The project was designated as JALPAC: Japanese Longitudinal Biomarker Study in PSP and CBD (UMIN18468).

Background: Progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) are progressive neurodegenerative disorders that are neuropathologically characterized by accumulation of phosphorylated 4-repeat tau in brain. Early diagnosis of PSP and CBD remains challenging, and the definite diagnosis is frequently deferred. The biomarkers for PSP and CBD have not been established.

Methods: The JALPAC cohort will comprise patients clinically diagnosed as having PSP or CBD followed longitudinally and comprehensively for biomarker assessment using standardized data acquisition protocols at 35 clinical sites with expertise in Japan. Clinical assessment and MRI study are performed. Biological samples including blood (plasma and serum), CSF, genomic DNA, and lymphoblastoid cell lines are stored at the JALPAC biorepository.

Results: To date (2015/12/24), 85 participants have been registered in JALPAC; the most frequent clinical phenotype is PSP-Richardson’s syndrome (n=31) followed by CBD/CBS (n=19) PSP-Parkinsonism (n=9), PSP-pure akinesia with gait freezing (n=6), and PSP-cerebellar ataxia (n=4).

Conclusions: The JALPAC project is expected to provide progress in developing biomarkers and understanding the natural history of PSP and CBD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/a-longitudinal-observational-study-of-a-cohort-of-patients-with-pspcbd-the-jalpac-project/