Safety and feasibility of autologous bone marrow cellular therapy in relapsing-progressive multiple sclerosis.
In this phase I study, we assessed the safety and feasibility of intravenous, autologous bone marrow (BM) cell therapy, without immunosuppressive preconditioning, in six patients with clinically definite, relapsing-progressive multiple sclerosis (MS). Assessment of efficacy was a secondary objective and employed clinical disability rating scales, multimodal evoked potential (MMEP) recordings, and magnetic resonance imaging (MRI) scans. Cells were harvested, filtered and infused intravenously in a day-case procedure that was well tolerated by patients and was not associated with any serious adverse events (AEs). Over a period of 12 months after the therapy, clinical disability scores showed either no change (Extended Disability Status Score, EDSS) or improvement (MS impact scale-29, MSIS-29), and MMEPs showed neurophysiological improvement. MRI scans did not show any significant changes over a post-therapy period of 3 months. The lack of serious adverse effects and the suggestion of a beneficial effect in this small sample of patients with progressive disease justify conducting a larger phase II/III study to make a fuller assessment of the efficacy of mobilization of autologous BM in patients with MS.
Rice
C M
CM
Institute of Clinical Neurosciences, University of Bristol, Frenchay Hospital, Bristol, UK.
Mallam
E A
EA
Whone
A L
AL
Walsh
P
P
Brooks
D J
DJ
Kane
N
N
Butler
S R
SR
Marks
D I
DI
Scolding
N J
NJ
D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods*; D004185:Disability Evaluation; D005071:Evoked Potentials; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods*; D008297:Male; D008875:Middle Aged; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000601:surgery*; D011446:Prospective Studies; D014182:Transplantation, Autologous