Stem-cell therapy for amyotrophic lateral sclerosis

Vincenzo Silani, Lidia Cova, Massimo Corbo, Andrea Ciammola, Elio Polli

Research output: Contribution to journalArticle

101 Citations (Scopus)

Abstract

Context With the lack of effective drug treatments for amyotrophic lateral sclerosis (ALS), and compelling preclinical data, stem-cell research has highlighted this disease as a candidate for stem-cell treatment. Stem-cell transplantation is an attractive strategy for neurological diseases and early successes in animal models of neurodegnerative disease generated optimism about restoring function or delaying degeneration in human beings. The restricted potential of adult stem cells has been challenged over the past 5 years by reports on their ability to acquire new unexpected fates beyond their embryonic lineage (transdifferentiation). Therefore, autologous or allogeneic stem cells, undifferentiated or transdifferentiated and manipulated epigenetically or genetically, could be a candidate source for local or systemic cell-therapies in ALS. Starting point Albert Clement and colleagues (Science 2003; 302: 113-17) showed that in SOD1G93A chimeric mice, motorneuron degeneration requires damage from mutant SOD1 acting in non-neuronal cells. Wild-type non-neuronal (glial) cells could delay degeneration and extend survival of mutant-expressing motorneurons. LetiziaMazzini and colleagues (Amyotroph Lateral Scler Other Motor Neuron Disord 2003; 4: 158-61) injected autologous bone-marrow-derived stem cells into the spinal cord of seven ALS patients. These investigators reported that the procedure had a reasonable margin of clinical safety. Where next? The success of cell-replacement therapy in ALS will depend a lot on preclinical evidence, because of the complexity and precision of the pattern of connectivity that needs to be restored in degenerating motoneurons. Stem-cell therapy will need to be used with other drugs or treatments, such as antioxidants and/or infusion of trophic molecules.

Original languageEnglish
Pages (from-to)200-202
Number of pages3
JournalLancet
Volume364
Issue number9429
DOIs
Publication statusPublished - Jul 10 2004

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Amyotrophic Lateral Sclerosis
Cell- and Tissue-Based Therapy
Stem Cells
Motor Neurons
Stem Cell Research
Animal Disease Models
Adult Stem Cells
Aptitude
Stem Cell Transplantation
Neuroglia
Pharmaceutical Preparations
Spinal Cord
Therapeutics
Antioxidants
Bone Marrow
Research Personnel
Safety
Survival

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Stem-cell therapy for amyotrophic lateral sclerosis. / Silani, Vincenzo; Cova, Lidia; Corbo, Massimo; Ciammola, Andrea; Polli, Elio.

In: Lancet, Vol. 364, No. 9429, 10.07.2004, p. 200-202.

Research output: Contribution to journalArticle

Silani, Vincenzo ; Cova, Lidia ; Corbo, Massimo ; Ciammola, Andrea ; Polli, Elio. / Stem-cell therapy for amyotrophic lateral sclerosis. In: Lancet. 2004 ; Vol. 364, No. 9429. pp. 200-202.
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