Biomarkers and Stem Cell-based Therapeutics in Alzheimer’s Disease: Recent Advances and Future Directions

Introduction: Alzheimer’s Disease (AD) is a progressive neurodegenerative disorder characterized by amyloid-β deposition, tau hyperphosphorylation, mitochondrial dysfunction, and synaptic loss, leading to cognitive and behavioral impairment. Despite extensive research, disease-modifying therapies remain limited. Recent advances in genetics, biomarker discovery, immunotherapy, and stem cell research have reshaped the understanding of AD pathophysiology and therapeutic opportunities. This review aims to summarize recent advances in Alzheimer’s disease pathogenesis, emerging fluid and imaging biomarkers, and stem cell–based therapeutic strategies, highlighting their integrated role in early diagnosis, disease monitoring, and precision medicine. Methods: A comprehensive literature search was conducted using PubMed, Scopus, and Web of Science databases. Peer-reviewed articles published in English focusing on AD genetics, biomarkers, immunotherapies, and stem cell–based interventions were included. Preclinical and clinical studies relevant to diagnosis, prognosis, and therapeutic development were critically analyzed. Results: Key biomarkers, including cerebrospinal fluid amyloid-β1-42, total tau, phosphorylated tau (p-Tau181), and neurofilament light chain, demonstrate high diagnostic and prognostic value. Advances in neuroimaging further enhance disease characterization. While CSF biomarkers remain the diagnostic gold standard, blood-based biomarkers offer promising, minimally invasive alternatives. Concurrently, stem cell therapies have shown neuroprotective, anti-inflammatory, and regenerative effects in preclinical AD models, supporting their potential role in disease modification. Conclusion: The convergence of biomarker-guided diagnostics and stem cell-based therapeutic strategies represents a promising direction for precision medicine in Alzheimer’s disease. Although translational challenges remain, integrating these approaches may improve early detection, disease progression monitoring, and the development of personalized therapeutic interventions.