Emerging therapeutic approaches in amyotrophic lateral sclerosis: From gene therapy and stem cells to autophagy and nanotechnology

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Background Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive motor neuron loss. This review summarizes recent advances in therapeutic strategies, with particular emphasis on gene therapy, stem cell approaches, and modulation of autophagy. Methods Recent literature on established and emerging therapeutic approaches for ALS was reviewed, focusing on disease-modifying therapies, gene-based interventions, stem cell therapies, autophagy modulation, and related translational developments. Results Recent literature highlights both established disease-modifying treatments, such as riluzole, edaravone, and tofersen, and emerging approaches, including antisense oligonucleotides, RNA interference, genome editing, stem cell transplantation, and autophagy-enhancing compounds. These strategies target key pathogenic mechanisms, including genetic mutations, neuroinflammation, and protein aggregation. While conventional pharmacological therapies offer limited benefit, novel molecular and cellular interventions demonstrate potential to modify disease progression. Gene therapies target mutation-specific pathways, stem cell-based therapies promote neuroprotection and immune modulation, and autophagy inducers aim to clear misfolded proteins. Despite encouraging preclinical and early clinical outcomes, challenges remain in delivery efficiency, disease heterogeneity, and biomarker development. Conclusion Therapeutic development in ALS is shifting toward precision medicine. Integration of personalized interventions and validated biomarkers is essential to improve treatment efficacy and patient outcomes.