Brain Injury
Oral Presentation
Steven Cramer, MD
Professor, Department of Neurology
University of California, Los Angeles
Los Angeles, California
Igor Semeniv, MD
Chief Doctor
Clinical Hospital
Kyiv, Kyyiv
Peter McAllister, MD
Medical Director
New England Institute for Neurology and Headache
STAMFORD, Connecticut
Satoshi Ikeda, MD PhD
Associate Professor
Department of Rehabilitation, Hokkaido University Hospital
Sapporo,, Hokkaido
Benjamin Frishberg, MD
Director of Clinical Research
Research Center of Southern California
Carlsbad, California
Albert Lai, MD
Medical Director
Westview Clinical Research
Rancho Mirage, California
Yusuke Shinoda, MD, PhD
Assistant Professor
Department of Rehabilitation Medicine, The University of Tokyo Hospital
Bunkyo-Ku, Tokyo
Alan Weintraub, MD, FACRM
Medical Director Brain Injury Program
Craig Hospital
englewood, Colorado
Michael Munin, MD
Professor
UPMC Department of PM&R
Pittsburgh, Pennsylvania
Neil Schwartz, MD, PhD
Clinical Professor
Stanford University
Palo Alto, California
Damien Bates, MD, PhD, FRACS, MBA
Chief Medical Officer & Head of Research
SanBio, Inc.
East Melbourne, Victoria
Intracranial implantation of SB623 cells was safe and well tolerated in patients with chronic TBI, and was associated with statistically significant improvement in motor status at 24 weeks.
Objective : To determine if intracranial implantation of SB623 cells can improve chronic motor deficits secondary to traumatic brain injury (TBI).
Design : A Phase 2, randomized, double-blind, surgical sham-controlled study (NCT02416492). Pre-specified 24-week interim analysis results are reported.
Setting : Patients were enrolled at 18 hospital centers in the US, Japan, and Ukraine.
Participants (or Animals, Specimens, Cadavers) : 61 patients (SB623=46, sham controls=15) with stable chronic motor deficits secondary to TBI.
Interventions : Allogeneic modified bone marrow-derived mesenchymal stem cells (SB623) were implanted via stereotactic intracranial injection. Patients were randomly stratified using a 1:1:1:1 ratio (2.5x106, 5.0x106, 10x106 SB623 cells, or surgical sham).
Main Outcome Measure(s) : Fugl-Meyer Motor Scale score (FMMS) change from baseline to 24 weeks, comparing SB623-treated patients versus controls.
Results : Treatment with SB623 cells was safe, with no dose-limiting toxicities or deaths. There was no significant difference in rate of treatment-emergent adverse events between groups (P=0.25). Serious adverse events were seen in five (10.9%) SB623-treated patients (two pre-operatively) versus three (20%) controls. In the modified intent-to-treat population of randomized patients completing surgery (N=61), FMMS score change from baseline to Week 24 was significantly greater for the combined SB623 group (n=46) compared to controls (n=15) (8.7±1.5 versus 2.4±2.7 points, P=0.04, least square mean±SE). The 5.0x106 SB623 group (n=15) experienced greatest FMMS score improvement at Week 24 (11.9±2.3 points), also significantly greater than controls (P=0.006). Significantly more SB623-treated patients than controls achieved the FMMS clinically meaningful threshold change of ≥10 points at Week 24 (39.1% versus 6.7%, P=0.04), a threshold that was achieved by 53.3% of the 5.0x106 SB623 group (P=0.015 versus control).
Conclusions : Intracranial implantation of SB623 was safe and well tolerated in patients with chronic TBI, and was associated with statistically significant improvement in motor status at 24 weeks.
