Whether it is a standard of care that is practical but not often appreciated or an emerging treatment that can change the landscape of a devastating disease, neurology as a whole is fueled by hope, and that is a gift that keeps on giving.
Jill Farmer, DO, MPH
THE HOLIDAY SEASON IS UPON US. It is a season of giving, gratitude, and, above all, hope. That is also the theme of this issue of NeurologyLive®. Our featured articles look at chronic neurologic conditions and the pipeline of progress that can offer hope to millions of patients or reestablish evidence-based practice to put into practical clinical use. With that in mind, it is important to reflect on the gift of progress and advancement that has been evident throughout this year in neurology.
Although the latest and greatest medication therapies are always sought after, it is important to remember that medicine is an art and a science, and sometimes, appreciating the art of therapy is most beneficial. For example, cognitive behavioral therapy continues to be a validated and effective treatment for all aspects of insomnia, from sleep hygiene to anxiety, and sometimes both physicians and patients need to be reminded of this. This is a theme to keep in mind across medicine.
In this issue, James F. Howard Jr, MD, and Nicholas Silvestri, MD, FAAN, share their insights into the current and future novel agents for myasthenia gravis. Myasthesia gravis includes multiple subtypes, each with unique needs and treatment strategies, and this discussion between the 2 experts outlines these algorithms in current practice, but also highlights what is to come in patient populations such as pregnant women and children.
Also in this issue is a highlight of Mayo Clinic, which has been a pioneer in the care of autoimmune disorders. Since the discovery of aquaporin-4 (AQP4) just 20 years ago, investigators in the field have not slowed down, and the neuroimmunology research laboratory discovers approximately 2 new antibody biomarkers of autoimmune or paraneoplastic neurological disorders per year. This translates into better quantitative tools for research, testing, and diagnosis, which ultimately leads to better treatment protocols rooted in evidence. The story of the discovery of the AQP4 biomarker and how it revolutionized neuromyelitis optica treatment is a tale of translational science at its best.
Lastly, this issue highlights a major moment for amyotrophic lateral sclerosis (ALS). With great revelry and fanfare, AMX0035 (Relyvrio; Amylyx Pharmaceuticals), was approved by the FDA in late 2022 as a disease-modifying therapy for ALS. In a study which randomly assigned 137 patients with ALS, and an open-label extension that included 90 patients on drug, safety and tolerability were demonstrated and a primary end point with an improvement on a standard scale—the ALS Functional Rating Scale-Revised—and a more than 6-month improvement in survival was met. This issue’s cover story provides a deeper look into this approval and what it could mean for the field heading into 2023.
Chronic neurological conditions are complex, and sometimes what we can offer as management feels incomplete. But what this issue amplifies is that whether it is a standard of care that is practical but not often appreciated or an emerging treatment that can change the landscape of a devastating disease, neurology as a whole is fueled by hope, and that is a gift that keeps on giving.