Emerging therapeutic strategies in multiple sclerosis: a focus on innovative and targeted approaches

Multiple sclerosis (MS) is an immune-mediated disease of the central nervous system marked by damage to myelin and nerve cells. Current treatments help control inflammation but have limited success in progressive stages and repairing nerve damage. This review aims to summarize new therapies for MS that target both inflammation and neurodegeneration beyond traditional immunosuppressive drugs. We conducted a thorough literature search to summarize key findings from original studies, including clinical trials investigating novel MS treatments such as Bruton's tyrosine kinase (BTK) inhibitors, CAR T-cell therapy, vaccines targeting Epstein-Barr virus (EBV) and specific antigens, drugs promoting remyelination, monoclonal antibodies, stem cell therapy, sphingosine-1-phosphate receptor modulators, cytokine blockers, gut microbiome interventions, and nanotechnology-based drug delivery. Emerging therapeutic strategies in MS increasingly target mechanisms beyond conventional immunosuppression, including B-cell and microglial modulation, immune tolerance induction, remyelination, cytokine signaling, microbiome regulation, and enhanced central nervous system drug delivery. BTK inhibitors and S1P receptor modulators demonstrated anti-inflammatory activity in clinical trials, although some agents failed to show superiority over established therapies. Cell-based therapies, including CAR T-cell therapy and stem cell transplantation, showed early promise in refractory disease but remain limited by safety concerns and insufficient long-term data. Remyelination-promoting agents and EBV-targeted immunotherapies demonstrated encouraging preliminary findings; however, many approaches remain in preclinical or early-phase clinical stages. Nanotechnology-based delivery systems and microbiome-directed therapies represent emerging areas with potential translational relevance. Emerging therapies in MS reflect a growing shift toward mechanism-based and potentially personalized therapeutic strategies. Although several approaches demonstrate promising preclinical and early clinical results, many remain investigational, and further large-scale studies are required to establish long-term efficacy, safety, and clinical applicability.