Cellular and Molecular Life Sciences

, Volume 71, Issue 17, pp 3257–3268 | Cite as

Stem cell transplantation for amyotrophic lateral sclerosis: therapeutic potential and perspectives on clinical translation

  • Irene Faravelli
  • Giulietta Riboldi
  • Monica Nizzardo
  • Chiara Simone
  • Chiara Zanetta
  • Nereo Bresolin
  • Giacomo P. Comi
  • Stefania CortiEmail author


Amyotrophic lateral sclerosis (ALS) is a fatal neurological disease characterized by degeneration of upper and lower motor neurons. There are currently no clinically impactful treatments for this disorder. Death occurs 3–5 years after diagnosis, usually due to respiratory failure. ALS pathogenesis seems to involve several pathological mechanisms (i.e., oxidative stress, inflammation, and loss of the glial neurotrophic support, glutamate toxicity) with different contributions from environmental and genetic factors. This multifaceted combination highlights the concept that an effective therapeutic approach should counteract simultaneously different aspects: stem cell therapies are able to maintain or rescue motor neuron function and modulate toxicity in the central nervous system (CNS) at the same time, eventually representing the most comprehensive therapeutic approach for ALS. To achieve an effective cell-mediated therapy suitable for clinical applications, several issues must be addressed, including the identification of the most performing cell source, a feasible administration protocol, and the definition of therapeutic mechanisms. The method of cell delivery represents a major issue in developing cell-mediated approaches since the cells, to be effective, need to be spread across the CNS, targeting both lower and upper motor neurons. On the other hand, there is the need to define a strategy that could provide a whole distribution without being too invasive or burdened by side effects. Here, we review the recent advances regarding the therapeutic potential of stem cells for ALS with a focus on the minimally invasive strategies that could facilitate an extensive translation to their clinical application.


Amyotrophic lateral sclerosis Stem cells Transplantation Clinical translation 



This work was supported by the ALS foundation to S.C. We also thank the ‘Associazione Amici del Centro Dino Ferrari’ for support.


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Copyright information

© Springer Basel 2014

Authors and Affiliations

  • Irene Faravelli
    • 1
  • Giulietta Riboldi
    • 1
  • Monica Nizzardo
    • 1
  • Chiara Simone
    • 1
  • Chiara Zanetta
    • 1
  • Nereo Bresolin
    • 1
  • Giacomo P. Comi
    • 1
  • Stefania Corti
    • 1
    Email author
  1. 1.Department of Pathophysiology and Transplantation (DEPT), Neuroscience Section, Dino Ferrari Centre, Neurology UnitUniversity of Milan, IRCCS Foundation Ca’Granda Ospedale Maggiore PoliclinicoMilanItaly

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