Bridging the Gap Between Science and Practice: The Project BIG Biorepository
Anna Tomczak1, Yamuna Joseph1, Julia Sumera1, Tara Tripathi Sarkar1, Safiyyah Bachar1, Crystal Ton-Nu1, Esther Nie1, Danwei Wu1, Jamie McDonald1, Lucas Kipp1, Christopher Lock1, May Han1, Jeffrey Dunn1
1Stanford University
Objective:
To create a comprehensive, multidisciplinary biorepository of human tissue from patients with Multiple Sclerosis (MS) and related neuroimmune diseases.
Background:
From the assumptions that MS primarily affects humans, that its immunopathology manifests often episodically, and that its confounding heterogeneity might be limited by longitudinal sample collection within an individual, we built a biorepository from sample collections of blood, cerebrospinal fluid (CSF), and stool. Specimens are annotated by disease state, clinical data and patient reported outcomes (PROs) to enable the translational investigation of MS.
Design/Methods:
Blood, CSF, and stool were longitudinally collected from participants, prioritizing times of change – including relapsing or remitted state or change in disease modifying immunotherapy. PROs on pain, quality of life (QoL), fatigue, anxiety/depression, and sleepiness were collected. Biospecimens and data were shared across the bedside-to-bench spectrum with bench scientists. Here we characterize the Project BIG MS cohort.
Results:
The biorepository has 491 participants and is composed of 5 disease groups: MS (64%), Neuromyelitis Optica (4%), Myelin oligodendrocyte glycoprotein antibody-associated disease (7%), Transverse Myelitis (2%), and Autoimmune Encephalitis (2%). We found a significant difference in sex and race between disease groups – with MS affecting predominantly Caucasian females (2 female:1 male). MS participants had subthreshold or no depression (mean = 5.74), mild anxiety (mean = 4.74), moderate QoL (score: 50-55), moderate fatigue (mean = 31.5) and moderate pain (mean = 12.65). There was a significant (p < 0.013) difference in pain scores, with MS participants scoring lower than the other groups. Within the MS cohort, 416 blood, 13 CSF, and 69 stool samples were collected. This inclusive cohort has led to 9 collaborations – 3 resulting in high-impact journal publications including the journals Nature and Science.
Conclusions:
By creating a multidisciplinary biorepository with multi-modal clinical correlates and time-associated PROs, the “Project BIG” initiative has shown promise in progressing MS research.