Abstract Details

Title Bridging the Gap Between Science and Practice: The Project BIG Biorepository

Topic Multiple Sclerosis

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 6-009

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.

Authors/Disclosures
Anna J. Tomczak, MSc (Stanford) PRESENTER	Miss Tomczak has nothing to disclose.
Yamuna Joseph (Stanford University)	No disclosure on file
Julia Sumera (Stanford Neurology)	Miss Sumera has nothing to disclose.
Tara Tripathi Sarkar (Stanford University)	No disclosure on file
Safiyyah Bachar (Stanford University)	No disclosure on file
Crystal Ton-Nu (Stanford University School of Medicine)	Crystal Ton-Nu has nothing to disclose.
Esther H. Nie, PhD (Stanford Healthcare Center for Academic Medicine (CAM))	Dr. Nie has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Nie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Medidata, Acorn AI. The institution of Dr. Nie has received research support from Roche/Genentech.
Danwei Wu, MD (Stanford University)	Dr. Wu has nothing to disclose.
Jamie C. McDonald, MD (Stanford University)	Dr. McDonald has nothing to disclose.
Lucas Kipp, MD	The institution of Dr. Kipp has received research support from Genentech.
Christopher Lock, MD, MBBS, PhD (Stanford University)	Dr. Lock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Lock has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amgen. Dr. Lock has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Turning Point Litigation. Dr. Lock has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Goodell, DeVries, Leech & Dann, LLP. Dr. Lock has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for McQuaide Blasko Attorneys at Law. Dr. Lock has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for BENNETT BIGELOW & LEEDOM P.S. .
May Han, MD (Stanford University)	Dr. Han has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Han has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arena Pharmaceuticals.
Jeffrey E. Dunn, MD, FAAN (Stanford University Medical Center)	Dr. Dunn has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Dunn has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genzyme. The institution of Dr. Dunn has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Progentec Diagnostics. Dr. Dunn has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyverna Therapeutics. Dr. Dunn has received intellectual property interests from a discovery or technology relating to health care.

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