# Therapeutic Potential of Mesenchymal Stem Cell-Derived Exosomes for Treatment of Contusive Spinal Cord Injury

> **NIH VA I01** · VA CONNECTICUT HEALTHCARE SYSTEM · 2020 · —

## Abstract

Project Summary/Abstract:
Spinal cord injury (SCI) is a major health care issue in our Veteran population. While rehabilitation medicine
provides for some therapeutic improvement for SCI there are no medical interventions that lead to protection of
neural tissue or induce regenerative responses. However, a number of cell therapy and small molecule
approaches to both protect and promote regeneration have been successful in pre-clinical studies using animal
models. Intravenous infusions of bone marrow-derived mesenchymal/stromal stem cells (MSCs) have been
demonstrated to produce beneficial effects in repairing tissue damage. Intravenous infusion of MSCs also has
beneficial effects in a number of experimental neurological disorders including stroke and spinal cord injury. In
some injury or disease models the therapeutic effects of MSC transplantation have been replicated by
transplantation of exosomes produced by these MSCs. Exosomes are nanosphere membrane bound particles
containing proteins and mRNAs/miRNAs which are highly stable and are capable of being taken up by other
cells and altering their function. These recently characterized secreted cellular nanospheres are believed to
play an important role in cell-cell communication in both health and disease. Preliminary studies in our lab
have found that intravenous infusion of exosomes produced by cultured MSCs can replicate therapeutic effects
of MSC infusion in spinal cord injured rats including improved locomotor function and decreased vascular
leakage in the spinal cord within one week after intravenous infusion. We propose to determine if these
beneficial effects observed early in SCI are sustained and how they compare to MSC infusion. The overall
objective of this project is to evaluate the therapeutic potential of intravenously delivered MSC-derived
exosomes for spinal cord injury (SCI). Exosomes are more stable and storable than cells and pose no risk of
aneuploidy and have a lower possibility of immune rejection. The preclinical work we propose here is directed
to provide basic information on safety and the potential therapeutic effects of intravenously delivered MSC-
derived exosomes in the context of potential future clinical studies in SCI patients. We currently have ongoing
clinical trials in both SCI and stroke using autologous MSCs, and success with the proposed studies could
facilitate the development of a clinical study using MSC-derived exosomes for SCI.

## Key facts

- **NIH application ID:** 9859326
- **Project number:** 5I01BX003190-04
- **Recipient organization:** VA CONNECTICUT HEALTHCARE SYSTEM
- **Principal Investigator:** Jeffery D. Kocsis
- **Activity code:** I01 (R01, R21, SBIR, etc.)
- **Funding institute:** VA
- **Fiscal year:** 2020
- **Award amount:** —
- **Award type:** 5
- **Project period:** 2017-01-01 → 2020-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9859326

## Citation

> US National Institutes of Health, RePORTER application 9859326, Therapeutic Potential of Mesenchymal Stem Cell-Derived Exosomes for Treatment of Contusive Spinal Cord Injury (5I01BX003190-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9859326. Licensed CC0.

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