Alzheimer’s disease (AD) is the most prevalent neurodegenerative disease, characterized by the accumulation of amyloid β-plaques and neurofibrillary tangles, oxidative stress, and mitochondrial dysfunction, among other pathological features. Current pharmacological treatments mainly provide symptoms relief, with limited efficacy on preventing/reversing disease progression. Therefore, novel disease-modifying therapies targeting these pathophysiological mechanisms may offer promising directions for advancing treatment. In this context, stem cell- and nerve growth factor-based therapies emerge as promising therapeutic approaches to bridge the gap in AD treatment, potentially supporting neuronal repair and slowing disease progression. Stem cells, including embryonic, induced pluripotent, mesenchymal, and neural stem cells, can generate new neurons to replace those lost or damaged during disease progression. Alternatively, they can be differentiated into glial cells to protect neurons from ongoing neuronal degeneration. Neurotrophic factors, endogenous proteins vital for neuronal survival, maintenance, and repair, activate repair genes involved in neuroprotection and regeneration. They are categorized into neurotrophins (e.g., nerve growth factor, brain-delivered growth factor, neurotrophin-3, or neurotrophin-4/5), transforming growth factor-β superfamily (e.g., glial derived neurotrophic factor, neurturin, artemin, persephin, and bone morphogenic protein family), and neuropoietic cytokines (e.g., ciliary neurotrophic factor, leukemia inhibitory factor, interleukin-6, oncostatin-M, and cardiotrophin-1). Overall, these therapies may potentially restore neural function and mitigate/prevent neuronal degeneration, representing promising therapeutic strategies. However, further research will allow to overcome the current challenges posed by stem cell- and neurotrophic factor-based therapies, ensuring their effective and safe application in clinical settings.