Astrocyte-based cell therapy: new hope for amyotrophic lateral sclerosis patients?

Amyotrophic lateral sclerosis (ALS) is a fatal paralytic disease with no cure or treatment to stop disease progression. Because ALS represents an urgent unmet medical need, a significant number of therapeutics are being tested in preclinical and clinical studies. A recent publication in Stem Cell Research & Therapy by Izrael and colleagues reports about embryonic stem cell-derived astrocytes as a potential cell therapy for ALS. Such cells behave as highly trophic “young astrocytes”, being able to delay disease onset and prolong survival when injected intrathechally in murine models of ALS overexpressing the SOD1(G93A) mutation. The safety and therapeutic potential of these cells are currently being evaluated in a clinical trial in ALS patients. This commentary discusses the mechanisms of action and potential therapeutic effects of these “young astrocytes” in ALS.