Furthering the Conversation of Treatment Personalization in NMOSD: Tammy Smith, MD, PhD

WATCH TIME: 4 minutes

"Fatigue that is not minor to them... if we overlook that our patients are too fatigued to take care of their kids, we're missing a huge opportunity to take care of our patients."

More than a decade ago, the discovery of aquaporin-4 (AQP4) led to the rapid drug development for patients with neuromyelitis optica spectrum disorder (NMOSD), a rare, demyelinating disorder of the central nervous system. The first approved therapy, eculizumab (Soliris; Alexion) came in 2019, followed by others, including satralizumab (Enspryng; Genentech) and inebilizumab (Uplizna; Horizon). More recently, in 2024, the FDA approved ravulizumab (Ultomiris; Alexion), another complement C5 inhibitor, as the latest treatment for those living with the condition.

In addition to these monoclonal antibodies, patients can also turn to other treatments such as rituximab (Rituxan) and immunosuppressives, such as azathioprine, mycophenolate, methotrexate, cyclophosphamide, and tocilizumab. Intravenous immunoglobulins, also known as antibodies, have also been shown to decrease the relapse rate of neuromyelitis optica. As the treatment landscape for NMOSD has expanded, the conversations around personalization and improving therapy selection have gained more attention.

Tammy Smith, MD, PhD, an assistant professor in the neurology department at the University of Utah in Salt Lake City, recently sat down with NeurologyLive® to discuss the topic of precision medicine for patients with NMOSD. While current treatments are based on biomarkers like AQP4 for seropositive patients, she stressed the need for further research into biomarkers for seronegative patients and the variability within AQP4-positive patients. In addition, Smith emphasized the importance of considering quality of life factors, such as fatigue, which can significantly affect patients despite treatment progress.