Stem cells therapy for ALS

Letizia Mazzini, Angelo Vescovi, Roberto Cantello, Maurizio Gelati, Alessandro Vercelli

Research output: Contribution to journalArticlepeer-review

18 Scopus citations


Introduction: Despite knowledge on the molecular basis of amyotrophic lateral sclerosis (ALS) having quickly progressed over the last few years, such discoveries have not yet translated into new therapeutics. With the advancement of stem cell technologies there is hope for stem cell therapeutics as novel treatments for ALS.Areas covered: We discuss in detail the therapeutic potential of different types of stem cells in preclinical and clinical works. Moreover, we address many open questions in clinical translation.Expert opinion: SC therapy is a potentially promising new treatment for ALS and the need to better understand how to develop cell-based experimental treatments, and how to implement them in clinical trials, becomes more pressing. Mesenchymal stem cells and neural fetal stem cells have emerged as safe and potentially effective cell types, but there is a need to carry out appropriately designed experimental studies to verify their long-term safety and possibly efficacy. Moreover, the cost-benefit analysis of the results must take into account the quality of life of the patients as a major end point. It is our opinion that a multicenter international clinical program aime d at fine-tuning and coordinating transplantation procedures and protocols is mandatory.

Original languageEnglish (US)
Pages (from-to)187-199
Number of pages13
JournalExpert Opinion on Biological Therapy
Issue number2
StatePublished - Feb 1 2016


  • Amyotrophic lateral sclerosis
  • Neuroinflammation
  • Neuroprotection
  • Stem cell

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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