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- W2536449589 abstract "OBJECTIVE: To investigate whether neural repair in MS can be detected by CSF biomarker analysis.BACKGROUND: We are currently conducting a Phase I FDA-approved trial to evaluate safety, tolerability, and preliminary efficacy of three IT administrations of autologous MSC-NPs in progressive MS. However, biomarkers that reflect neural repair and CNS regeneration are currently lacking. CSF Fetuin-A has been identified as a biomarker for disease activity in MS, showing significantly elevated levels in patients with active disease. Similarly, elevated CSF levels Neurofilament Light (NF-L) have been correlated with axonal damage in MS. Finally, Hepatocyte Growth Factor (HGF) is a pleiotropic factor proposed as a mechanism of action in the regenerative effects of MSCs. In this study, we measured the CSF concentrations of each of these proteins and compared them to clinical outcomes.DESIGN/METHODS: Ten of the 20 enrolled patients in the ongoing trial have received three MSC-NP doses. CSF was taken at each treatment, as well as 3 months after the final treatment. Cell-free CSF was stored at -80°C until use. Fetuin-A, NF-L, and HGF levels were measured by ELISA. Clinical assessment of treatment outcomes was determined by EDSS, timed 25 foot walk, PEG test, PASAT, evoked potentials, and urodynamics testing. CSF analysis in all 20 patients will be analyzed at study completion.RESULTS: Following MSC-NP treatment, 3/10 patients showed a significant reduction in CSF NF-L and 2/10 patients showed a significant increase in CSF HGF. The only patient who showed statistically significant changes from baseline in both HGF (38[percnt] increase) and in NF-L (28[percnt] decrease) also demonstrated a 1 point improvement in EDSS with quantifiable functional recovery post-treatment. Fetuin-A levels showed no consistent trends.CONCLUSIONS: These results indicate that HGF and NF-L may represent quantifiable CSF biomarkers of neural repair that correlate with improvements elicited by treatment. Disclosure: Dr. Tuddenham has nothing to disclose. Dr. Harris has nothing to disclose. Dr. Sadiq has nothing to disclose." @default.
- W2536449589 created "2016-10-28" @default.
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- W2536449589 date "2016-04-05" @default.
- W2536449589 modified "2023-09-22" @default.
- W2536449589 title "Effects of Intrathecal MSC-Derived Neural Progenitors (MSC-NP) Treatment on Biomarker Levels in the CSF in Patients with Progressive Multiple Sclerosis (P3.066)" @default.
- W2536449589 hasPublicationYear "2016" @default.
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