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Translational stem cell therapy for amyotrophic lateral sclerosis

Nature Reviews Neurology volume 8, pages 172–176 (2012)Cite this article

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Abstract

Effective treatments are urgently needed for amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease characterized by the loss of motor neurons. In 2009, the FDA approved the first phase I safety trial of direct intraspinal transplantation of neural stem cells into patients with ALS, which is currently in progress. Stem cell technologies represent a promising approach for treating ALS, but several issues must be addressed when translating promising experimental ALS therapies to patients. This article highlights the key research that supports the use of stem cells as a therapy for ALS, and discusses the rationale behind and approach to the phase I trial. Completion of the trial could pave the way for continued advances in stem cell therapy for ALS and other neurodegenerative diseases.

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Figure 1: Accurate anatomical targeting of stem cell delivery.
Figure 2: Spinal cord stabilization and injection systems for intraspinal stem cell transplantation.

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Acknowledgements

We thank M. Marsala, M. Hefferan, J. Rile, B. Raore and J. Taub for their technical assistance with the preclinical studies. Preclinical studies were funded in part by the A. Alfred Taubman Medical Research Institute, the ALS Association and Neuralstem. N. Boulis invented the stem cell delivery device used in the phase I trial. The phase I trial is funded by Neuralstem, and we acknowledge the valuable input of K. Johe and the Data Safety Monitoring Board of the trial. We are grateful to the Emory ALS Center, patients and families for their participation.

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Authors and Affiliations

  1. Department of Neurosurgery, Emory University, 1365-B Clifton Road NE, Suite 2200, Atlanta, 30322, GA, USA

    Nicholas M. Boulis & Thais Federici

  2. Department of Neurology, Emory University, 101 Woodruff Circle, WMB Suite 6000, Atlanta, 30322, GA, USA

    Jonathan D. Glass

  3. Department of Neurology, University of Michigan, 109 Zina Pitcher Place, 5017 AAT-BSRB, Ann Arbor, 48109-2200, MI, USA

    J. Simon Lunn, Stacey A. Sakowski & Eva L. Feldman

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  1. Nicholas M. Boulis
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Correspondence to Eva L. Feldman.

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Competing interests

N. M. Boulis invented the device utilized in the trial for the safe and accurate injection of stem cells into the human spinal cord. He received an inventor fee and retains rights to royalty payments on distribution of the device. Exclusive licensure for this technology has been purchased by NeuralStem. N. M. Boulis, T. Federici and J. D. Glass received funding from NeuralStem for the phase I trial. The other authors declare no competing interests.

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Boulis, N., Federici, T., Glass, J. et al. Translational stem cell therapy for amyotrophic lateral sclerosis. Nat Rev Neurol 8, 172–176 (2012). https://doi.org/10.1038/nrneurol.2011.191

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