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A Phase III Clinical Trial Showing Limited Efficacy of Autologous Mesenchymal Stem Cell Therapy for Spinal Cord Injury

Oh, Sun Kyu MD; Choi, Kyoung Hyo MD, PhD; Yoo, Jong Yoon MD, PhD; Kim, Dae Yul MD, PhD; Kim, Sang Joon MD, PhD; Jeon, Sang Ryong MD, PhD

doi: 10.1227/NEU.0000000000001056
Research-Human-Clinical Trials

BACKGROUND: In our previous report, 3 of 10 patients with spinal cord injury who were injected with autologous mesenchymal stem cells (MSCs) showed motor improvement in the upper extremities and in activities of daily living.

OBJECTIVE: To report on the results of a phase III clinical trial of autologous MSCs therapy.

METHODS: Patients were selected based on the following criteria: chronic American Spinal Injury Association B status patients who had more than 12 months of cervical injury, and no neurological changes during the recent 3 months of vigorous rehabilitation. We injected 1.6 × 107 autologous MSCs into the intramedullary area at the injured level and 3.2 × 107 autologous MSCs into the subdural space. Outcome data were collected over 6 months regarding neurological examination, magnetic resonance imaging with diffusion tensor imaging, and electrophysiological analyses.

RESULTS: Among the 16 patients, only 2 showed improvement in neurological status (unilateral right C8 segment from grade 1 to grade 3 in 1 patient and bilateral C6 from grade 3 to grade 4 and unilateral right C8 from grade 0 to grade 1 in 1 patient). Both patients with neurological improvement showed the appearance of continuity in the spinal cord tract by diffusion tensor imaging. There were no adverse effects associated with MSCs injection.

CONCLUSION: Single MSCs application to intramedullary and intradural space is safe, but has a very weak therapeutic effect compared with multiple MSCs injection. Further clinical trials to enhance the effect of MSCs injection are necessary.

ABBREVIATIONS: ADL, Activities of Daily Living

ASIA, American Spinal Injury Association

BM, bone marrow

DTI, diffusion tensor imaging

EP, electrophysiological studies

KMFDS, Korean Ministry of Food and Drug Safety

MEP, motor evoked potential

MSC, mesenchymal stem cell

SCI, spinal cord injury

SEP, somatosensory evoked potential

Departments of *Neurological Surgery,

Rehabilitation Medicine, and

§Radiology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea

Correspondence: Sang Ryong Jeon, MD, PhD, Department of Neurological Surgery, Asan Medical Center, University of Ulsan College of Medicine, 88, Olympic-ro 43-gil, SongPa-Gu, Seoul 138-736, Republic of Korea. E-mail: srjeon@amc.seoul.kr

Received March 11, 2015

Accepted August 22, 2015

Copyright © by the Congress of Neurological Surgeons