Complete Spinal Cord Injury Treatment using Autologous Bone Marrow Cell Transplantation and Bone Marrow Stimulation with Granulocyte Macrophage-Colony Stimulating Factor (GM-CSF): Phase I/II Clinical Trial

¹ Inha Neural Repair Center, Department of Neurosurgery, Inha University College of Medicine, Incheon, Korea.
² Inha Neural Repair Center, Inha Research Institute for Medical Sciences, Inha University College of Medicine, Incheon, Korea.
³ Inha Neural Repair Center, Inha University College of Medicine, Incheon, Korea. Anesthesiology, Ajou University Hospital, Suwon, Korea
⁴ Diagnostic Pathology, Ajou University Hospital, Suwon, Korea
⁵ Rehabilitation medicine, Ajou University Hospital, Suwon, Korea
⁶ Inha Neural Repair Center, Inha University College of Medicine, Incheon, Korea. Physiology, Ajou University Hospital, Suwon, Korea, Center for Advanced Medical Education by BK21 Project, Inha University College of Medicine, Incheon, Korea.
⁷ Department of Orthopedics, Ajou University Hospital, Suwon, Korea
⁸ Department of Neurosurgery, Inha University College of Medicine, Incheon, Korea.
⁹ Inha Neural Repair Center, Department of Neurosurgery, Center for Advanced Medical Education by BK21 Project, Inha University College of Medicine, Incheon, Korea.

^* To whom correspondence should be addressed. E-mail: hayoon{at}inha.ac.kr.

	Abstract

To assess the safety and therapeutic efficacy of autologous BMCs transplantation and the administration of GM-CSF, a Phase I/II open-label and non-randomized study was conducted on 35 complete SCI patients. The BMCs was transplanted by injection into the surround of spinal cord injury within 14 injury days (n=17), between 14 days to 8 weeks (n=6) and at more than 8 weeks (n=12) after injury. In the control group, all patients (n=13) were treated only with conventional decompression and fusion surgery without BMCs transplantation. The patients underwent pre-operative and follow-up neurological assessment using American Spinal Injury Association standards (AIS), electrophysiological monitoring and magnetic resonance imaging (MRI). The mean follow-up period was 10.4 months after injury. At 4 month, the MRI analysis showed the enlargement of spinal cords and the small enhancement of the cell implantation sites, which were not any adverse lesions such as malignant transformation, hemorrhage, new cysts or infections. Furthermore, the BMCs transplantation and GM-CSF administration were not associated with any serious adverse clinical events increasing morbidities. The AIS grade increased in 30.4% of the acute and subacute treated patients (AIS A to B or C), while, no significant improvement was observed in the chronic treatment group. Increasing neuropathic pain during the treatment and tumor formation at the site of transplantation are still remaining to be concerned. Long-term and large scale multi-center clinical study is required to determine its precise therapeutic effect.