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 Corti

Research output: Contribution to journalArticle

21 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)3257-3268
Number of pages12
JournalCellular and Molecular Life Sciences
Volume71
Issue number17
DOIs
Publication statusPublished - 2014

Fingerprint

Amyotrophic Lateral Sclerosis
Stem Cell Transplantation
Motor Neurons
Cell- and Tissue-Based Therapy
Stem Cells
Central Nervous System
Therapeutics
Neuroglia
Respiratory Insufficiency
Glutamic Acid
Oxidative Stress
Inflammation

Keywords

  • Amyotrophic lateral sclerosis
  • Clinical translation
  • Stem cells
  • Transplantation

ASJC Scopus subject areas

  • Cell Biology
  • Molecular Biology
  • Molecular Medicine
  • Pharmacology
  • Cellular and Molecular Neuroscience

Cite this

Stem cell transplantation for amyotrophic lateral sclerosis : Therapeutic potential and perspectives on clinical translation. / Faravelli, Irene; Riboldi, Giulietta; Nizzardo, Monica; Simone, Chiara; Zanetta, Chiara; Bresolin, Nereo; Comi, Giacomo P.; Corti, Stefania.

In: Cellular and Molecular Life Sciences, Vol. 71, No. 17, 2014, p. 3257-3268.

Research output: Contribution to journalArticle

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