Advances in maintenance therapies for neuromyelitis optica spectrum disorders: A new era of targeted drugs.

BACKGROUND

Neuromyelitis optica spectrum disorder (NMOSD) is a rare inflammatory demyelinating disease of the central nervous system, mainly affecting the optic nerve and spinal cord, with a high rate of recurrence and disability. The spectrum of possibilities for NMOSD maintenance therapies has increased recently due to the development of novel medications based on a deeper understanding of its pathogenesis.

METHODS

Clinical and preclinical advances in NMOSD maintenance therapies were searched in PubMed up until July 20, 2024.

RESULTS

We reviewed the treatment options including approved monoclonal antibodies, clinical trial drugs, and preclinical targets. Both randomized clinical trials and real-word studies have evaluated the safety and efficacy of monoclonal antibodies against complement, IL-6 receptor and B cells. Drugs in the early stage of clinical trials are mainly focused on small molecule targeted drugs and cell-based therapies. Additionally, preclinical studies aimed to explore new targets, such as aquaporin blocking antibodies, based on experiences in cell cultures and animal models.

CONCLUSIONS

From biologics to small molecules and cells, researchers have endeavored to provide better medical care for NMOSD patients. With the emergence of more and more targeted drugs, it is anticipated that the treatment of NMOSD will enter a new era.