A Pilot Study to Assess microRNA Biomarkers in Early and Later Stage Multiple Sclerosis

Multiple Sclerosis is a chronic autoimmune disorder of the central nervous system. While
there are current immunomodulatory therapies that have been shown to be efficacious in the
early stages of MS, these therapies are less potent in the later phases of MS. We can look
at magnetic resonance imaging with gadolinium to measure active disease but it does not
detect axonal degeneration. Therefore, there is a need to identify other biomarkers that may
be used to diagnose MS and predict disease progression. Biomarkers found in the
cerebrospinal fluid (CSF) are in closer proximity to the inflammatory lesion sites and are
more sensitive.

This pilot study seeks to characterize differences in microRNA profiles and cell products in
the early and later stages of MS, with the hope that that microRNA profiles could then be
correlated to clinical and CSF inflammation indexes. CSF will be obtained from 45
participants.

Inclusion Criteria:

- able to understand and agree to informed consent;

- male or female patients 18-68 years of age

- no disease modifying therapy 60 days prior to Baseline

- EDSS score of less than or equal to 3.0 if a CIS patient; or less than or equal to
7.5 if SPMS patient

- Labs within normal range no greater than 2 times the ULN or at the discretion of the
PI

- weight 46 kilograms to 127 kilograms inclusive

- no active systemic infection

- not currently pregnant or breast feeding

- no history of corticosteroid treatment or relapse within 60 days prior to Baseline.

Exclusion Criteria:

- not able to understand informed consent

- if any of the inclusion criteria is not met

- HIV infection, or current active Hepatitis B or C infection, positive HCVAb or HBsAG
or HBcAB

- positive pregnancy test

- patient withdraws consent

- Coumadin use within 60 days prior to Baseline