Efficacy and safety of exosomes from Wharton’s Jelly-derived mesenchymal stem cells in traumatic brain injury

BACKGROUND Traumatic brain injury (TBI) is a significant public health issue, leading to long-term neurological impairments. Current treatments offer limited recovery, particularly in restoring lost functions. Mesenchymal stem cell-derived exosomes (MSCdE) have shown potential for promoting neuroprotection and regeneration. This study evaluates the safety and efficacy of MSCdE therapy in TBI patients. AIM To evaluate the safety and efficacy of MSCdE therapy in TBI patients. METHODS Five patients (mean age 27.00 ± 4.06 years) with TBI from combat injuries were treated with six rounds of MSCdE therapy (3 mL intrathecally and 3 mL intramuscularly per round). The patients were followed for one year. Adverse events were assessed using the Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0), and functional outcomes were evaluated with the functional independence measure (FIM), Modified Ashworth Scale (MAS), and Karnofsky Performance Scale (KPS). RESULTS No serious adverse events occurred, and only mild side effects [subfebrile fever (37.5-37.9 ℃), pain] were reported (CTCAE Grade 1). FIM motor scores improved significantly (46.20 ± 16.39 to 64.20 ± 18.20, P < 0.01), and FIM cognitive scores also showed significant improvement (30.60 ± 4.56 to 34.00 ± 1.41, P < 0.001). While MAS scores improved (right/left: 4.60/3.60 to 2.20/1.60), these changes were not statistically significant (P > 0.05), possibly due to low baseline spasticity. KPS scores significantly improved (46.00 ± 11.40 to 72.00 ± 8.37, P < 0.001), indicating enhanced overall functional status and quality of life. CONCLUSION MSCdE therapy is safe and effective in improving motor function, cognition, and quality of life in TBI patients. Larger, controlled trials are needed to further validate these findings and optimize MSCdE therapy for TBI treatment.