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Article first published online: 15 MAY 2012
Copyright © 2012 AlphaMed Press
Volume 30, Issue 6, pages 1144–1151, June 2012
How to Cite
Glass, J. D., Boulis, N. M., Johe, K., Rutkove, S. B., Federici, T., Polak, M., Kelly, C. and Feldman, E. L. (2012), Lumbar Intraspinal Injection of Neural Stem Cells in Patients with Amyotrophic Lateral Sclerosis: Results of a Phase I Trial in 12 Patients. STEM CELLS, 30: 1144–1151. doi: 10.1002/stem.1079
Author contributions: J.D.G.: site PI and primary writer of the manuscript; N.M.B.: surgeon; K.J.: sponsor of trial and developer of stem cell product; S.B.R.: responsible for EIM data; T.F.: protocol development and stem cell biologist; M.P.: lead clinical research nurse; C.K.: lead clinical research coordinator; E.L.F.: protocol development and overall PI.
Disclosure of potential conflicts of interest is found at the end of this article.
First published online in STEM CELLSEXPRESS March 13, 2012.
- Issue published online: 15 MAY 2012
- Article first published online: 15 MAY 2012
- Accepted manuscript online: 13 MAR 2012 02:23PM EST
- Manuscript Accepted: 20 FEB 2012
- Manuscript Received: 28 DEC 2011
- NeuralStem, Inc
- Neuralstem, Inc
- Amyotrophic lateral sclerosis;
- Motor neuron disease;
- Stem cells;
- Spinal cord;
- Clinical trial
Advances in stem cell biology have generated intense interest in the prospect of transplanting stem cells into the nervous system for the treatment of neurodegenerative diseases. Here, we report the results of an ongoing phase I trial of intraspinal injections of fetal-derived neural stems cells in patients with amyotrophic lateral sclerosis (ALS). This is a first-in-human clinical trial with the goal of assessing the safety and tolerability of the surgical procedure, the introduction of stem cells into the spinal cord, and the use of immunosuppressant drugs in this patient population. Twelve patients received either five unilateral or five bilateral (10 total) injections into the lumbar spinal cord at a dose of 100,000 cells per injection. All patients tolerated the treatment without any long-term complications related to either the surgical procedure or the implantation of stem cells. Clinical assessments ranging from 6 to 18 months after transplantation demonstrated no evidence of acceleration of disease progression due to the intervention. One patient has shown improvement in his clinical status, although these data must be interpreted with caution since this trial was neither designed nor powered to measure treatment efficacy. These results allow us to report success in achieving the phase I goal of demonstrating safety of this therapeutic approach. Based on these positive results, we can now advance this trial by testing intraspinal injections into the cervical spinal cord, with the goal of protecting motor neuron pools affecting respiratory function, which may prolong life for patients with ALS. STEM CELLS2012;30:1144–1151